Relevant bibliographies by topics / Personalized gene therapy

Academic literature on the topic 'Personalized gene therapy'

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Published: 14 March 2023

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Journal articles on the topic "Personalized gene therapy"

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Schaly, Sabrina, Merry Ghebretatios, and Satya Prakash. "Baculoviruses in Gene Therapy and Personalized Medicine." Biologics: Targets and Therapy Volume 15 (April 2021): 115–32. http://dx.doi.org/10.2147/btt.s292692.

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Harris, Tim. "Gene and drug matrix for personalized cancer therapy." Nature Reviews Drug Discovery 9, no. 8 (August 2010): 660. http://dx.doi.org/10.1038/nrd3181-c1.

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Kohno, Takashi, Koji Tsuta, Katsuya Tsuchihara, Takashi Nakaoku, Kiyotaka Yoh, and Koichi Goto. "RETfusion gene: Translation to personalized lung cancer therapy." Cancer Science 104, no. 11 (October 1, 2013): 1396–400. http://dx.doi.org/10.1111/cas.12275.

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Iacobas, Sanda, and Dumitru Andrei Iacobas. "Personalized 3-Gene Panel for Prostate Cancer Target Therapy." Current Issues in Molecular Biology 44, no. 1 (January 15, 2022): 360–82. http://dx.doi.org/10.3390/cimb44010027.

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Many years and billions spent for research did not yet produce an effective answer to prostate cancer (PCa). Not only each human, but even each cancer nodule in the same tumor, has unique transcriptome topology. The differences go beyond the expression level to the expression control and networking of individual genes. The unrepeatable heterogeneous transcriptomic organization among men makes the quest for universal biomarkers and “fit-for-all” treatments unrealistic. We present a bioinformatics procedure to identify each patient’s unique triplet of PCa Gene Master Regulators (GMRs) and predict consequences of their experimental manipulation. The procedure is based on the Genomic Fabric Paradigm (GFP), which characterizes each individual gene by the independent expression level, expression variability and expression coordination with each other gene. GFP can identify the GMRs whose controlled alteration would selectively kill the cancer cells with little consequence on the normal tissue. The method was applied to microarray data on surgically removed prostates from two men with metastatic PCas (each with three distinct cancer nodules), and DU145 and LNCaP PCa cell lines. The applications verified that each PCa case is unique and predicted the consequences of the GMRs’ manipulation. The predictions are theoretical and need further experimental validation.
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Barthélémy, Florian, and Nicolas Wein. "Personalized gene and cell therapy for Duchenne Muscular Dystrophy." Neuromuscular Disorders 28, no. 10 (October 2018): 803–24. http://dx.doi.org/10.1016/j.nmd.2018.06.009.

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Sahoo, Firoj Kumar. "Enalapril and the VEGFA gene: Personalized medicine in hypertension therapy." European Journal of Clinical Pharmacology 72, no. 1 (November 23, 2015): 125. http://dx.doi.org/10.1007/s00228-015-1984-y.

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BERTOLOTTI, ROGER. "EDITORIAL: "AUTOLOGOUS STEM CELL GENE THERAPY: TOWARD A UNIVERSAL PLATFORM FOR PERSONALIZED THERAPY"." Gene Therapy and Regulation 03, no. 01 (March 2007): 1–14. http://dx.doi.org/10.1142/s1568558607000022.

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Dugo, Ketty, Francesca Bruno, Valentina Sturiale, Desiree Brancato, Salvatore Saccone, and Concetta Federico. "Hereditary Transthyretin-Related Amyloidosis: Genetic Heterogeneity and Early Personalized Gene Therapy." Biomedicines 10, no. 10 (September 25, 2022): 2394. http://dx.doi.org/10.3390/biomedicines10102394.

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Point mutations of the transthyretin (TTR) gene are related with hereditary amyloidosis (hATTR). The number of people affected by this rare disease is only partially estimated. The real impact of somatic mosaicism and other genetic factors on expressivity, complexity, progression, and transmission of the disease should be better investigated. The relevance of this rare disease is increasing and many efforts have been made to improve the time to diagnosis and to estimate the real number of cases in endemic and non-endemic areas. In this context, somatic mosaicism should be better investigated to explain the complexity of the heterogeneity of the hATTR clinical features, to better estimate the number of new cases, and to focus on early and personalized gene therapy. Gene therapy can potentially improve the living conditions of affected individuals and is one of the central goals in research on amyloidosis related to the TTR gene, with the advantage of overcoming liver transplantation as the sole treatment for hATTR disease.
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M, Mavroudi, Zarogoulidis P, Porpodis K, Kioumis I, Lampaki S, Yarmus L, Malecki R, Zarogoulidis K, and Malecki M. "Stem cells’ guided gene therapy of cancer: New frontier in personalized and targeted therapy." Journal of Cancer Research & Therapy 2, no. 1 (January 1, 2014): 22–33. http://dx.doi.org/10.14312/2052-4994.2014-4.

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NAKAMURA, Tomonori, and Koujirou YAMAMOTO. "2. Application of Gene Analysis and TDM in Personalized Drug Therapy." Rinsho yakuri/Japanese Journal of Clinical Pharmacology and Therapeutics 44, no. 4 (2013): 361–62. http://dx.doi.org/10.3999/jscpt.44.361.

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Dissertations / Theses on the topic "Personalized gene therapy"

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Patsali, Petros. "Advanced personalized gene therapy of B-thalassaemia." Thesis, King's College London (University of London), 2018. https://kclpure.kcl.ac.uk/portal/en/theses/advanced-personalized-gene-therapy-of-bthalassaemia(4e7d967d-eec8-4663-ae40-bd36b1793a71).html.

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Thalassaemias are amongst the commonest single‐gene disorders worldwide, but offer only limited curative treatment choices. Autologous transplantation of gene‐therapy‐corrected cells is therefore investigated by numerous groups and is already under clinical trials for β‐thalassaemia, based on gene addition by lentiviral vectors (LVs). The main aim of this project was the development of personalised gene therapy (GT) for β‐thalassaemia caused by the common and severe HBBIVSI‐110 mutation, which results in missplicing of intron 1. The resulting aberrant mRNA interferes with protein expression from normal endogenous or vector‐encoded HBB. The two therapy approaches taken were a) modification or co‐transduction of the LV ΜΑ821_ΗΒΒ (“GLOBE”) to achieve concurrent vector‐derived HBB expression and RNAi‐based suppression of aberrant RNA and b) development of designer nucleases for genome editing and functional correction of the HBBIVSI‐110 mutation. To establish proof of principle for both approaches we developed transgenic murine erythroleukaemia cell lines MEL MA821‐HBBIVSI‐110 and MEL MA821‐HBBNormal with on average two vector copies per genome (VCN) and an additional, clonal cell line of VCN=1 for MEL MA821 HBBIVSI‐110. Approach a) Depletion of aberrant HBBIVSI‐110 mRNA might enhance translation of vector‐derived or residual endogenous HBB mRNA and could be achieved by HBBIVSI‐110‐specific RNAi, mediated by expression of lentivirally delivered shRNAs. To this end, four different HBBIVSI‐110 mRNA‐specific shRNAs were cloned into the U6‐promoter‐driven pLKO.I lentiviral vector (LV). In clonal MEL HBBIVSI‐110 cells, two constructs, pLKO.I shIVSI‐110 Mid & Mid2, exhibited no discernible change of normal:aberrant HBB mRNA but a significant increase of HBB chains relative to untransduced samples. Importantly, our approach was then validated by transduction of HBB IVSI‐110‐patient‐derived hCD34+ cells, in which we observed a ~30% increase of HBB:HBA chain ratios in samples transduced with shIVSI‐110 MID, regardless of co‐transduction with other vectors. These data indicate that RNAi‐targeting of aberrant HBB mRNAs, if not therapeutic in its own right, could substantially improve efficiency of GT by HBB gene augmentation and could thus lower conditioning, VCN and gene‐expression requirements for LV‐based gene therapies. Approach b) As an alternative to gene augmentation we established a novel genome‐editing approach in which permanent functional correction the HBBIVSI‐110 mutation was achieved with the targeted disruption of the aberrant SA site and/or its sequence context. This approach, based on the efficient non‐homologous end‐joining (NHEJ) repair pathway, used HBBIVSI‐110 ‐ specific designer nucleases, transcription activator‐like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 RNA‐guided endonuclease (RGENs). Assessment of targeted disruption efficiency of our designer nucleases on gDNA or episomal reporter plasmids indicated the superiority of the HBB TALEN over RGEN for HBBIVSI‐110. Plasmid transfection of HBBIVSI‐110 specific ‐ designer nucleases into MEL MA821‐HBBIVSI‐110 transgenic cells resulted in functional correction at the RNA (RT‐qPCR) and protein level (Immunoblots). In addition, full characterisation (type, frequency and context) of induced insertions/deletions (INDELs) was achieved by T7 Endonuclease 1 assay, decomposition of sequence traces (TIDE) and Sanger sequencing of TOPO clones from edited bulk cells. Using edited MEL MA821‐HBBIVSI‐110 clones we moreover correlated specific INDELs with HBB expression at the RNA and protein level, which confirmed our hypothesis that functional correction of splicing could be achieved by disruption of upstream sequences of the aberrant SA site, leaving intact the HBBIVSI‐110 mutation. Subsequent validation of our nucleases in patient‐derived CD34+ cells by electroporation of HBB TALEN L1/R1 plasmid DNA was impaired by low targeted disruption (12%) despite high‐transfection efficiencies. Even though a marginal increase in the ΗΒΒ:ΗΒΑ chain ratio was detected, analyses of mRNA levels were inconclusive, calling for additional experimentation in CD34+ cells with more appropriate expression systems. Overall, proof of principle was established for NHEJ‐based functional repair of HBBIVSI‐110, and we suggest that the approach can be adapted for the functional correction of other of disease‐causing gain‐of‐function mutations in non‐coding regulatory regions.
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KATIA, CAPITANI. "Genome editing for clinically relevant mutations in genetic diseases and cancer." Doctoral thesis, Università di Siena, 2022. http://hdl.handle.net/11365/1211914.

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The present thesis concerns of two sections. The first one focuses on the application of Cluster Regularly Interspaced Short Palindromic Repeats (CRISPR) system as a tool for precise genome targeting and genome editing; the association between specific endonuclease and RNA guides complementary to the DNA target allows its targeting with single-nucleotide precision. CRISPR/Cas is able to perform Double-Strand Breaks (DSBs) at a target site which are soon repaired by cellular repairing mechanism, non-homologous end joining (NHEJ) or homology-directed repair (HDR). The first part of my project aims to explore and demonstrate the efficacy of a personalized therapeutic approach based on the CRISPR/Cas9 technology associated with adeno-associated viral vectors (AAVs)s, a mutation-specific gene therapy to restore mutated genes in genetic diseases to their original sequence trough the HDR-mediated correction. I developed an mCherry/EGFP reporter cassette where the reporter gene bears a mutation-specific target. It connects the mCherry and the EGFP (out of frame) coding sequences. Due to a frameshift, the reactivation of the EGFP allows the visualization of cells in which Cas9 had targeted the mutation-specific sequence leading to the production of Indels. I worked to edit mutations involved in specific genetic diseases such as mutations in FOXG1 or in MECP2, which are responsible for Rett syndrome, in the IQSEC2 gene that causes an intellectual disability clinically related to the Rett syndrome and in COL4A3 and COL4A5 causing Alport syndrome. In the second part of my study, I worked on developing a gene editing system aims to selective targeting to cancer cells while preserving the genetic integrity of normal cells. To this aim, I plan to exploit microhomology-mediated end joining (MMEJ) through Cas12a, an RNA-directed endonuclease that causes double-strand breaks with staggered ends, to insert in-frame the Herpes Simplex Virus –Thymidine Kinase suicide gene to trigger cell death. I designed and developed a construct to target a patient-specific single nucleotide variant within a coding sequence of the TP53 gene, from a patient with Chronic Lymphocytic Leukemia characterized by clonal expansion of clones bearing this TP53 mutation. I am able to detect the proper integration of the suicide gene sequence by analyzing the treated cells by fluorescence-activated cell sorting (FACS). Indeed, a green fluorescent protein (EGFP) sequence is linked to the TK by a 2A peptide system, thus green fluorescent cells are also the one expressing for the TK gene. The second section of my thesis concerns the COVID-19 pandemic global crisis and the need to understand how best to study and treat COVID-19. A key focus is sharing and analyzing data to learn about the genetic determinants of COVID-19 susceptibility, severity, and outcomes. In particular, my work has been focused on the TLR7 gene that has been involved as an important pattern recognition receptor for the ssRNA of SARS-CoV-2. We demonstrate that rare loss-of-function variants in the TLR7 gene in young men with severe COVID-19 and with no prior history of major chronic diseases were associated with impaired TLR7 signaling and type I and II IFN responses.
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Förster, Susann [Verfasser], Hanspeter [Akademischer Betreuer] Herzel, Wolfgang [Akademischer Betreuer] Kemmner, and Reinhold [Akademischer Betreuer] Schäfer. "Gene expression profiling of human lymph node-positive gastric adenocarcinomas : towards personalized prognosis and therapy / Susann Förster. Gutachter: Hanspeter Herzel ; Wolfgang Kemmner ; Reinhold Schäfer." Berlin : Humboldt Universität zu Berlin, Mathematisch-Naturwissenschaftliche Fakultät I, 2011. http://d-nb.info/101497531X/34.

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Valls, Margarit Jordi. "Comprehensive identification and characterisation of germline structural variation within the Iberian population." Doctoral thesis, Universitat de Barcelona, 2021. http://hdl.handle.net/10803/673719.

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One of the central aims of biology and biomedicine has been the characterisation and understanding of genetic variation across humans, to answer important evolutionary questions and to explain phenotypic variability concerning the diseases. Understanding genetic variability, is key to study this relationship (through imputation and GWASs) and to translate the results into improved clinical protocols. Different initiatives have emerged around the world to systematically characterise the genetic variability of specific human populations from whole-genome sequences, usually by selecting geographical regions. Examples such as 1000 Genomes (1000G)1, GoNL2, HRC, UK10K3 or Estonian population4, have already identified and characterised millions of genetic variants across different populations. In combination with imputation analysis, these sequenced-based projects allow increasing the statistical power and resolution of Genome-Wide Association Studies (GWAS), identifying and discovering new disease-associated variants5. Additionally, genetic variability among population groups is associated with geographic ancestry and can affect the disease risk or treatment efficacy differently6,7. For this reason, population- specific reference panels are necessary to characterise their genetic diversity and to assess its effect on human phenotypes, improving GWAS studies, as one of the cornerstones of precision medicine7. Existing genetic variability panels include Single Nucleotide Variants (SNVs) and indels (<50bp) but are limited in large Structural Variants (SV) (≥50bp). Technical and methodological limitations hindered the discovery of SVs using Next-generation Sequencing (NGS) technologies, as it produced False-Discovery Rates between 9-89% and recall 10-70%, depending on the SV type and size8. On average, the genomic variation between two human genomes is around 0.1%, but this difference increases to 1.5% with SVs8. The SVs also affect 3-10 times more nucleotides than SNVs9 (4M SNVs per genome10), showing their potential effect on human phenotypes. For this reason, including a complete catalogue of SVs in reference panels will increase the power in GWAS studies and provide opportunities to find new disease-associated variants. To overcome these limitations, in this thesis, we have generated the first genome-wide Iberian haplotype reference panel, mainly focused on Structural Variants, using 785 samples whole-genome sequenced (WGS) at high coverage (30X) from the GCAT-Genomics for life project. We designed a complete strategy, including an extensive benchmarking of multiple variant calling programs and by building specific Logistic Regression Models (LRM) for SV types, as well as phasing strategies to come up with a high quality and comprehensive genetic variability panel. This strategy was benchmarked using different controlled sets of variants, showing high precision and recall values across all variant types and sizes. The application of this strategy to our GCAT whole-genome samples resulted in the identification of 35,431,441 genetic variants, classified as 30,325,064 SNPs, 5,017,19 small indels (< 50bp), and 89,178 larger SV (≥ 50bp). The latter group was further subclassified into 33,244 deletions, 6,269 duplications, 12,782 insertions, 10,115 inversions, 18,779 transposons and 7,989 translocations, covering all ranges of frequencies and sizes. Besides, 60% of the discovered SVs were not catalogued in any repository, thus increasing the insights of SV in humans. Additionally, 52.44% of common and 71.63% of low-frequency SVs were not included in any haplotype reference panel. Thus, new SVs could be used in GWAS, adding more value to the Iberian-GCAT catalogue. The prediction of the functional impact of the SVs shows that these variants might have a central role in several diseases. Of all SVs included in the Iberian-GCAT catalogue, 46% overlapped in genes (both protein-coding genes and non-protein-coding genes), highlighting their potential impact on human traits. Besides, 92.7% of protein-coding genes were located outside low-complexity (repeated) genomic regions, expecting short-reads from NGS to capture the most interpretable SVs in humans11. Moreover, 32.93% of SVs affected protein-coding genes with a predicted loss of function intolerance (pLI) effect, further supporting the potential implication of these variants on complex diseases and therefore enabling a better explanation of missing heritability. Importantly, taking advantage of high coverage (30X), we accurately determine the genotypes of SVs, enabling to phase together with SNVs and indels, and increasing the SV phasing accuracy, in contrast to 1000G and GoNL. Besides, high coverage allowed to use Phasing Informative Reads (PIRs), increasing the phasing performance. The overall strategy enables the community to expand and improve the imputation possibilities within GWAS. The Iberian-GCAT haplotype reference panel created in this thesis, imputes accurately common SVs, with near ~100% of agreement with sequencing results. Although the Iberian- GCAT haplotype reference panel can be used in all populations from different continental groups, due to closer ancestries, the imputation performance is high in European and Latin American populations, reflected in the amount of low-frequency (1% ≤ MAF < 5%) and rare (1% > MAF) variants imputed at high info scores. These results demonstrated the versatility of our resource, increasing their performance in closer ancestries. In general, we observed that when the allele frequency decreases, the imputation accuracy drops too, highlighting the necessity to include more samples in reference panels, to impute low-frequency and rare variants efficiently, which normally are expected to have more functional impact on diseases. Finally, we compared the imputation possibilities of the 1000G and GoNL reference panels, with our Iberian-GCAT reference panel. We observed that the Iberian-GCAT reference panel outperformed the imputation of high-quality SVs by 2.7 and 1.6-fold compared to 1000G and GoNL, respectively. Also, the overall imputation quality is higher, showing the value of this new resource in GWAS as it includes more SVs than previous reference panels. The combination of different reference panels will improve the resolution and statistical power of GWAS, thus increasing the chances to find more risk variants in complex diseases, and ultimately, translated this insight to precision medicine.
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Vahedi, Golnaz. "An Engineering Approach Towards Personalized Cancer Therapy." 2009. http://hdl.handle.net/1969.1/ETD-TAMU-2009-08-2941.

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Cells behave as complex systems with regulatory processes that make use of many elements such as switches based on thresholds, memory, feedback, error-checking, and other components commonly encountered in electrical engineering. It is therefore not surprising that these complex systems are amenable to study by engineering methods. A great deal of effort has been spent on observing how cells store, modify, and use information. Still, an understanding of how one uses this knowledge to exert control over cells within a living organism is unavailable. Our prime objective is "Personalized Cancer Therapy" which is based on characterizing the treatment for every individual cancer patient. Knowing how one can systematically alter the behavior of an abnormal cancerous cell will lead towards personalized cancer therapy. Towards this objective, it is required to construct a model for the regulation of the cell and utilize this model to devise effective treatment strategies. The proposed treatments will have to be validated experimentally, but selecting good treatment candidates is a monumental task by itself. It is also a process where an analytic approach to systems biology can provide significant breakthrough. In this dissertation, theoretical frameworks towards effective treatment strategies in the context of probabilistic Boolean networks, a class of gene regulatory networks, are addressed. These proposed analytical tools provide insight into the design of effective therapeutic interventions.
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Books on the topic "Personalized gene therapy"

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Olga, Golubnitschaja, ed. Predictive diagnostics and personalized treatment: Dream or reality. Hauppauge, NY: Nova Science Publishers, 2009.

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Genes, chromosomes, and disease: From simple traits, to complex traits, to personalized medicine. Upper Saddle River, New Jersey: FT Press Science, 2011.

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Smalley, Keiran. Current Challenges in Personalized Cancer Medicine. Elsevier Science & Technology Books, 2012.

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Smalley, Keiran. Current Challenges in Personalized Cancer Medicine. Elsevier Science & Technology Books, 2012.

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Bakhtiar, Syeda Marriam, and Erum Dilshad, eds. Omics Technologies for Clinical Diagnosis and Gene Therapy: Medical Applications in Human Genetics. BENTHAM SCIENCE PUBLISHERS, 2022. http://dx.doi.org/10.2174/97898150795171220101.

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Genetic disorders have been the focus of scientists for a long time. The emergence of next-generation sequencing techniques has ushered a new era in genetics and several developments have occurred in human genetics. The scientific perspective has also been widened with omics technologies that allow researchers to analyze genetic sequences and their expression products. An integrated approach is being used not only for diagnosis but also for disease management and therapeutic purposes. This book highlights emerging areas of omics technology and its application in the diagnosis and management of human genetic disorders. The book covers three areas of research and implementation: 1) Diagnosis (covering conventional strategies to next-generation platforms). This section focuses on the role of in silico analysis, databases and multi-omics of single-cell which will help in designing better management strategies. 2) Disease Management and therapeutic interventions. This section starts with genetic counselling and progresses to more specific techniques such as pharmacogenomics and personalized medicine, gene editing techniques and their applications in gene therapies and regenerative medicine. 3) Case studies. This section discusses the applications and success of all the above-mentioned strategies on selected human disorders. This book serves as a handy reference for students and academics studying advanced omics techniques in biochemistry and molecular genetics as part of courses in life sciences, pharmacology and medicine.
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Barker, Richard. The supply of new medicine—unlimited? Oxford University Press, 2013. http://dx.doi.org/10.1093/med/9780199600663.003.0002.

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Chapter 1 describes the supply of new medical technology which exploits the huge advances we are making in bioscience, the build-up a profound understanding of how the beautiful molecular machines of the living cell actually work, and how they link together in the almost unimaginably complex system that is our body, and asks what fresh pharmaceutical innovations are on the horizon? Will gene and cell therapy reach the diseases that drugs cannot reach? Is ‘Personalized medicine’ practical? What will converging technologies—therapeutics, diagnostics, informatics, nanotechnology—bring us?
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Fehse, Boris, Ferdinand Hucho, Sina Bartfeld, Stephan Clemens, Tobias Erb, Heiner Fangerau, Jürgen Hampel, et al., eds. Fünfter Gentechnologiebericht. Nomos Verlagsgesellschaft mbH & Co. KG, 2021. http://dx.doi.org/10.5771/9783748927242.

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In the ‘Fifth Gene Technology Report’, renowned experts provide an overview of current developments and their applications in the dynamically evolving research field of gene and biotechnologies. They examine, among other topics, genetic diagnostics, somatic gene therapy, the development of vaccines, stem cell and organoid research, green gene technology, synthetic biology, gene drives, genome editing, epigenetics and single cell analysis. In addition to reporting on the current state of affairs in this field, the authors also discuss society’s perception of gene technologies and ethical and legal issues relating to them, such as genome edit-ing, cerebral organoids and big data in personalised medicine. Moreover, the interdisciplinary task force ‘Gentechnologiebericht’ (Gene Technology Report) offers recommendations on action that could be taken in relation to the key issues. With contributions by Karla Alex, Sina Bartfeld, Meik Bittkowski, Inge Broer, Lorina Buhr, Stephan Clemens, Wolfgang Van den Daele, Hans-Georg Dederer, Tobias J. Erb, Nina Gasparoni, Heiner Fangerau, Boris Fehse, Jürgen Hampel, Louise Herde, Ferdinand Hucho, Ali Jawaid, Aida Khachatryan, Sarah Kohler, Alma Kolleck, Martin Korte, Cordula Kropp, Alfons Labisch, Markus Lehmkuhl, Melanie Leidecker-Sandmann, Annette Leßmöllmann, Isabelle M. Mansuy, Lilian Marx-Stölting, Andreas Merk, Yannick Milhahn, Fruzsina Molnár-Gábor, Stefan Mundlos, Staffan Müller-Wille, Angela Osterheider, Anja Pichl, Barbara Prainsack, Jens Reich, Marlen Reinschke, Ortwin Renn, Hans-Jörg Rheinberger, Arnold Sauter, Hannah Schickl, Silke Schicktanz, Volker Stollorz, Constanze Störk-Biber, Jochen Taupitz, Jörn Walter, Eva C. Winkler, Martin Zenke and Michael M. Zwick.
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Canli, Turhan. Neurogenethics. Edited by Turhan Canli. Oxford University Press, 2014. http://dx.doi.org/10.1093/oxfordhb/9780199753888.013.27.

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Ethical inquiry has followed advances in biology for decades, and different fields within biology have given rise to overlapping yet distinct areas of ethical inquiry. Genethics focuses on the ethics of genetics. Neuroethics focuses on the ethics of neuroscience. The author suggests that developments in molecular psychology, in which the tools of molecular biology are applied to study behavior, bring a new confluence of factors to generate a set of new questions, unique to the combination of neuroscience and genetics: neurogenethics—the ethics of neurogenetics. The questions are unique and novel because they emerge when genetic techniques are applied to the brain: to reprogram neural circuits and psychological processes, better predict behavior, personalize mental health treatment, and understand the Self. This chapter outlines the problem space and discusses specific examples of future neurogenethics research areas: viral-mediated gene therapy directed at the brain, optogenetics, imaging genetics, therapygenetics, and direct-to-consumer genomics.
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Book chapters on the topic "Personalized gene therapy"

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Baum, Bruce J. "Gene Therapy for Xerostomia." In Genomics, Personalized Medicine and Oral Disease, 333–43. Cham: Springer International Publishing, 2015. http://dx.doi.org/10.1007/978-3-319-17942-1_15.

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Greenberger, Joel S., Michael W. Epperly, Peter Wipf, Song Li, Valerian Kagan, and Xiang Gao. "Gene Therapy for Mucositis." In Genomics, Personalized Medicine and Oral Disease, 345–62. Cham: Springer International Publishing, 2015. http://dx.doi.org/10.1007/978-3-319-17942-1_16.

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Vorhies, John S., Donald D. Rao, Neil Senzer, and John Nemunaitis. "siRNA Versus shRNA for Personalized Cancer Therapy: Mechanisms and Applications." In Gene-Based Therapies for Cancer, 51–62. New York, NY: Springer New York, 2010. http://dx.doi.org/10.1007/978-1-4419-6102-0_4.

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Bhalla, Parinishtha, Anukriti Verma, Bhawna Rathi, Shivani Sharda, and Pallavi Somvanshi. "Exploring Molecular Signatures in Spondyloarthritis: A Step Towards Early Diagnosis." In Proceedings of the Conference BioSangam 2022: Emerging Trends in Biotechnology (BIOSANGAM 2022), 142–55. Dordrecht: Atlantis Press International BV, 2022. http://dx.doi.org/10.2991/978-94-6463-020-6_15.

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AbstractSpondyloarthritis is an acute inflammatory disorder of the musculoskeletal system often accompanied by pain, stiffness, bone and tissue damage. It majorly consists of ankylosing spondylitis, psoriatic arthritis and reactive arthritis. It follows a differential diagnosis pattern for demarcation between the spondyloarthritis subtypes and other arthritic subtypes such as rheumatoid arthritis, juvenile arthritis and osteoarthritis due to the heterogeneity causing gradual chronicity and complications. Presence of definite molecular markers can not only improve diagnosis efficiency but also aid in their prognosis and therapy. This study is an attempt to compose a refined list of such unique and common molecular signatures of the considered subtypes, by employing a reductionist approach amalgamating gene retrieval, protein-protein interaction network, functional, pathway, micro-RNA-gene and transcription factor-gene regulatory network analysis. Gene retrieval and protein-protein interaction network analysis resulted in unique and common interacting genes of arthritis subtypes. Functional annotation and pathway analysis found vital functions and pathways unique and common in arthritis subtypes. Furthermore, miRNA-gene and transcription factor-gene interaction networks retrieved unique and common miRNA’s and transcription factors in arthritis subtypes. Furthermore, the study identified important signatures of arthritis subtypes that can serve as markers assisting in prognosis, early diagnosis and personalized treatment of arthritis patients requiring validation via prospective experimental studies.
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Li, Yao, Lawrence Chan, Huy V. Nguyen, and Stephen H. Tsang. "Personalized Medicine: Cell and Gene Therapy Based on Patient-Specific iPSC-Derived Retinal Pigment Epithelium Cells." In Retinal Degenerative Diseases, 549–55. Cham: Springer International Publishing, 2015. http://dx.doi.org/10.1007/978-3-319-17121-0_73.

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Paro, Renato, Ueli Grossniklaus, Raffaella Santoro, and Anton Wutz. "Epigenetics and Cancer." In Introduction to Epigenetics, 151–77. Cham: Springer International Publishing, 2021. http://dx.doi.org/10.1007/978-3-030-68670-3_8.

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AbstractAlterations in chromatin function and epigenetic mechanisms are a hallmark of cancer. The disruption of epigenetic processes has been linked to altered gene expression and to cancer initiation and progression. Recent cancer genome sequencing projects revealed that numerous epigenetic regulators are frequently mutated in various cancers. This information has not only started to be utilized as prognostic and predictive markers to guide treatment decisions but also provided important information for the understanding of the molecular mechanisms of epigenetic regulation in both physiological and pathological conditions. Furthermore, the reversible nature of epigenetic aberrations has led to the emergence of the promising field of epigenetic therapy that has already provided new therapeutic options for patients with malignancies characterized by epigenetic alterations, laying the basis for new and personalized medicine.
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Grosios, Konstantina, Harald Petry, and Jacek Lubelski. "Adeno-Associated Virus Gene Therapy and Its Application to the Prevention and Personalised Treatment of Rare Diseases." In Rare Diseases, 131–57. Dordrecht: Springer Netherlands, 2014. http://dx.doi.org/10.1007/978-94-017-9214-1_9.

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Wilson, James M., and Nelson A. Wivel. "Gene Therapy." In Genomic and Personalized Medicine, 610–19. Elsevier, 2009. http://dx.doi.org/10.1016/b978-0-12-369420-1.00053-6.

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Deshpande, Madhura, Shravan Kumar Sriraman, and Vladimir Torchilin. "NANOTHERANOSTICS IN GENE THERAPY." In Nanotheranostics for Personalized Medicine, 191–221. WORLD SCIENTIFIC, 2016. http://dx.doi.org/10.1142/9789814713535_0008.

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Oliveira, Beatriz B., Alexandra R. Fernandes, and Pedro V. Baptista. "Nanotheranostics in Gene Therapy." In Advances in Cancer Nanotheranostics for Experimental and Personalized Medicine, 82–115. BENTHAM SCIENCE PUBLISHERS, 2020. http://dx.doi.org/10.2174/9789811456916120010007.

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Conference papers on the topic "Personalized gene therapy"

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Critelli, Rosina Maria, Fabiola Milosa, Barbara Lei, Luca Marzi, Rosario Condello, Elena Turola, Nicola De Maria, Antonino Maiorana, Gianluigi Giannelli, and Erica Villa. "Abstract A07: Gene analysis maps HCC heterogeneity and orientates personalized therapy." In Abstracts: AACR Special Conference on Developmental Biology and Cancer; November 30 - December 3, 2015; Boston, Massachusetts. American Association for Cancer Research, 2016. http://dx.doi.org/10.1158/1557-3125.devbiolca15-a07.

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Jurišić, Vladimir. "POSSIBILITIES OF CYTOKINE DETERMINATION AND THEIR ANALYSIS IN VARIOUS TISSUES." In 1st INTERNATIONAL Conference on Chemo and BioInformatics. Institute for Information Technologies, University of Kragujevac,, 2021. http://dx.doi.org/10.46793/iccbi21.089j.

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Cytokines are small proteins that participate in many interactions between cells of the immune system as well as between many tissue cells including tumors. Currently, there is no universal classification of cytokines and they can be analyzed based on the cells that produce them or based on the type of activity. Cytokines have been studied for many years in medicine firstly in cancer patients in serum, but also in many other diseases including inflammation or other autoimmune diseases or other pathological conditions. Cytokines are still being discovered, and for many of them the structure, biological action and genes responsible for their regulation have already been determined. Bearing in mind that the development of technology has been developing enormously in the last period and those new methods of cytokine determination in various fluids and micro-concentrations are available to us. Here, the aim is to focus on the specific possibilities of determination and analysis of cytokine values in different tissues including cell culture supernatants, in individual cells as well as their genetic regulation. However, to understand their complex action in biological systems, including the pleiotropic effect of cytokines showing some time the overlap in the actions various models of analysis and interpretation of the obtained data are recommended today. This is especially complex and problematic in recent times of understanding the cytokine gene regulation and especially the possibility of their prediction. To resolve these problems, numerous databases have been created on the previously available experimental data, although their connection is not yet very clear. In addition, using integration of data, it is expected predict some models and systems in a specific situation, although it is still very difficult. So, aims are predict values in definitive situation and compare with some standards. Therefore, new methods of interpretation and new programs for analysis have been created. We expect that based on the new possibilities of analysis, better results will be achieved and that the role of these mediators for individual or personalized diagnosis or therapy in biomedicine will be determined.
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Foy, Susan, Kyle Jacoby, Daniela Bota, Theresa Hunter, Adam Schoenfeld, Zheng Pan, Eric Stawiski, et al. "1478 A phase I study of personalized adoptive TCR T cell therapy in patients with solid tumors: safety, efficacy, and T cell trafficking to tumors of non-virally gene edited T cells." In SITC 37th Annual Meeting (SITC 2022) Abstracts. BMJ Publishing Group Ltd, 2022. http://dx.doi.org/10.1136/jitc-2022-sitc2022.1478.

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Nascimento, Renan Gomes do. "AN IN SÍLICO ANALYSIS DETECTED MEMBERS OF THE PLECKSTRIN HOMOLOGY-LIKE DOMAIN FAMILY B AS POTENTIAL PROGNOSTIC BIOMARKERS IN PATIENTS WITH BREAST CANCER." In Abstracts from the Brazilian Breast Cancer Symposium - BBCS 2021. Mastology, 2021. http://dx.doi.org/10.29289/259453942021v31s2034.

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Objectives: Despite advances in the molecular classification of breast cancer, our understanding of the pathophysiology of the disease is still limited mainly due to the considerable intratumoral heterogeneity. Thus, hundreds of other candidates for biomarkers are being investigated and studied for possible implications for diagnosis, prognosis, and personalized therapy. In this context, members of the Pleckstrin homology-like domain family B (PHLDB), which is composed of three genes located on different chromosomes: PHLDB1 (11q23.3), PHLDB2 (3q13.2), and PHLDB3 (19q13.3), are under investigation by different research groups as potential biomarkers in different types of cancer. It has been reported that the altered expression of these genes is involved in the tumorigenic process. In this study, we sought to understand the prognostic and predictive value of genes from the PHLDB family as potential biomarkers in breast oncology. Conclusions: Our findings provide new insights into the potential role of PHLDB family members as clinical predictors in breast cancer. Unlike what has already been described in the literature, it appears that members of the PHLDB family are potential tumor suppressor genes in breast cancer. Further clinical and experimental studies are needed to better understand the relationship between the expression of the members of the PHLDB family and the tumorigenic process of the breast and its prognostic and predictive values in breast cancer.
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Shi, Ivy, Lihong Yin, and Patrick C. Ma. "Abstract 4466: Integrative study of dysregulated genes in translocated ALK-positive non-small cell lung cancer and personalized targeted therapy resistance." In Proceedings: AACR 104th Annual Meeting 2013; Apr 6-10, 2013; Washington, DC. American Association for Cancer Research, 2013. http://dx.doi.org/10.1158/1538-7445.am2013-4466.

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Micheel, Christine M., Debayani Chakravarty, Jiaojiong Gao, Ian Maurer, Clinton Miller, Kenna R. Shaw, Mia A. Levy, and Nikolaus Schultz. "Abstract LB-104: Clinical actionability and clinical trial matching for GENIE patient genotypes using My Cancer Genome, Personalized Cancer Therapy, and OncoKB." In Proceedings: AACR Annual Meeting 2017; April 1-5, 2017; Washington, DC. American Association for Cancer Research, 2017. http://dx.doi.org/10.1158/1538-7445.am2017-lb-104.

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