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Journal articles on the topic 'Gene therapy; Genetic diseases'

Author: Grafiati
Published: 4 June 2021
Last updated: 1 February 2022

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1

Kohn, Donald B., W. French Anderson, and R. Michael Blaese. "Gene Therapy for Genetic Diseases." Cancer Investigation 7, no. 2 (January 1989): 179–92. http://dx.doi.org/10.3109/07357908909038283.

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2

Desnick, Robert J., and Edward H. Schuchman. "Gene therapy for genetic diseases." Pediatrics International 40, no. 3 (June 1998): 191–203. http://dx.doi.org/10.1111/j.1442-200x.1998.tb01912.x.

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3

Rasko AO, John. "Progress in gene therapy for genetic diseases." Pathology 48 (February 2016): S39. http://dx.doi.org/10.1016/j.pathol.2015.12.102.

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4

McIvor, R. Scott. "Gene therapy of genetic diseases and cancer." Pediatric Transplantation 3 (November 1999): 116–21. http://dx.doi.org/10.1034/j.1399-3046.1999.00050.x.

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5

Wilson, James M. "Genetic Diseases, Immunology, Viruses, and Gene Therapy." Human Gene Therapy 25, no. 4 (April 2014): 257–61. http://dx.doi.org/10.1089/hum.2014.2511.

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6

Wei, Chunjiang, Weijia Kong, and Zuhong He. "Application of gene therapy in auditory system diseases." STEMedicine 1, no. 1 (January 2, 2020): e17. http://dx.doi.org/10.37175/stemedicine.v1i1.17.

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How to prevent and treat auditory related diseases through genetic intervention is a hotspot in the field of hearing research in recent years. With the development of molecular biology, molecular genetics, genetic engineering, etc., especially, gene regulation has made a major breakthrough in the research of inner ear hair cell regeneration in recent years, which may provide us with a novel and efficient way to treat auditory related diseases. This review includes the latest research on gene therapy in hereditary deafness, drug deafness, aging-related hearing loss, and noise-related hearing loss.
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7

Davis, Lori, and Frank Park. "Gene therapy research for kidney diseases." Physiological Genomics 51, no. 9 (September 1, 2019): 449–61. http://dx.doi.org/10.1152/physiolgenomics.00052.2019.

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A resurgence in the development of newer gene therapy systems has led to recent successes in the treatment of B cell cancers, retinal degeneration and neuromuscular atrophy. Gene therapy offers the ability to treat the patient at the root cause of their malady by restoring normal gene function and arresting the pathological progression of their genetic disease. The current standard of care for most genetic diseases is based upon the symptomatic treatment with polypharmacy while minimizing any potential adverse effects attributed to the off-target and drug-drug interactions on the target or other organs. In the kidney, however, the development of gene therapy modifications to specific renal cells has lagged far behind those in other organ systems. Some positive strides in the past few years provide continued enthusiasm to invest the time and effort in the development of new gene therapy vectors for medical intervention to treat kidney diseases. This mini-review will systematically describe the pros and cons of the most commonly tested gene therapy vector systems derived from adenovirus, retrovirus, and adeno-associated virus and provide insight about their potential utility as a therapy for various types of genetic diseases in the kidney.
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8

Bunnell, Bruce A., and Richard A. Morgan. "Gene Therapy for Infectious Diseases." Clinical Microbiology Reviews 11, no. 1 (January 1, 1998): 42–56. http://dx.doi.org/10.1128/cmr.11.1.42.

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SUMMARY Gene therapy is being investigated as an alternative treatment for a wide range of infectious diseases that are not amenable to standard clinical management. Approaches to gene therapy for infectious diseases can be divided into three broad categories: (i) gene therapies based on nucleic acid moieties, including antisense DNA or RNA, RNA decoys, and catalytic RNA moieties (ribozymes); (ii) protein approaches such as transdominant negative proteins and single-chain antibodies; and (iii) immunotherapeutic approaches involving genetic vaccines or pathogen-specific lymphocytes. It is further possible that combinations of the aforementioned approaches will be used simultaneously to inhibit multiple stages of the life cycle of the infectious agent.
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9

Chaum, Edward, and Mark P. Hatton. "Gene Therapy for Genetic and Acquired Retinal Diseases." Survey of Ophthalmology 47, no. 5 (September 2002): 449–69. http://dx.doi.org/10.1016/s0039-6257(02)00336-3.

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10

Berger, Adeline, Séverine Maire, Marie-Claude Gaillard, José-Alain Sahel, Philippe Hantraye, and Alexis-Pierre Bemelmans. "mRNAtrans-splicing in gene therapy for genetic diseases." Wiley Interdisciplinary Reviews: RNA 7, no. 4 (March 28, 2016): 487–98. http://dx.doi.org/10.1002/wrna.1347.

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11

Emery, David W. "Gene therapy for genetic diseases: On the horizon." Clinical and Applied Immunology Reviews 4, no. 6 (October 2004): 411–22. http://dx.doi.org/10.1016/j.cair.2004.05.001.

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12

Poletti, Valentina, and Fulvio Mavilio. "Designing Lentiviral Vectors for Gene Therapy of Genetic Diseases." Viruses 13, no. 8 (August 2, 2021): 1526. http://dx.doi.org/10.3390/v13081526.

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Lentiviral vectors are the most frequently used tool to stably transfer and express genes in the context of gene therapy for monogenic diseases. The vast majority of clinical applications involves an ex vivo modality whereby lentiviral vectors are used to transduce autologous somatic cells, obtained from patients and re-delivered to patients after transduction. Examples are hematopoietic stem cells used in gene therapy for hematological or neurometabolic diseases or T cells for immunotherapy of cancer. We review the design and use of lentiviral vectors in gene therapy of monogenic diseases, with a focus on controlling gene expression by transcriptional or post-transcriptional mechanisms in the context of vectors that have already entered a clinical development phase.
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13

Servida, P., and C. Bordignon. "Gene Therapy and Apheresis." International Journal of Artificial Organs 16, no. 5_suppl (May 1993): 116–18. http://dx.doi.org/10.1177/039139889301605s23.

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Gene therapy is becoming a real therapeutic chance in some genetic disorders. The first candidates to this approach are genetic diseases which involve hematopoiesis, because of the facility for the collection and the manipulation of hematopoietic progenitors. Apheresis techniques, which are able to collect a great number of mononuclear cells from peripheral blood, are ideal for obtaining a large number of cells which can be transfected. Future uses of gene therapy techniques could be: the treatment of hematopoietic genetic disorders, procedures of gene marking, and the manipulation of normal hematopoietic cells with the objective of increasing their resistance to myelotoxic drugs.
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14

Roth, Theodore L., and Alexander Marson. "Genetic Disease and Therapy." Annual Review of Pathology: Mechanisms of Disease 16, no. 1 (January 24, 2021): 145–66. http://dx.doi.org/10.1146/annurev-pathmechdis-012419-032626.

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Genetic diseases cause numerous complex and intractable pathologies. DNA sequences encoding each human's complexity and many disease risks are contained in the mitochondrial genome, nuclear genome, and microbial metagenome. Diagnosis of these diseases has unified around applications of next-generation DNA sequencing. However, translating specific genetic diagnoses into targeted genetic therapies remains a central goal. To date, genetic therapies have fallen into three broad categories: bulk replacement of affected genetic compartments with a new exogenous genome, nontargeted addition of exogenous genetic material to compensate for genetic errors, and most recently, direct correction of causative genetic alterations using gene editing. Generalized methods of diagnosis, therapy, and reagent delivery into each genetic compartment will accelerate the next generations of curative genetic therapies. We discuss the structure and variability of the mitochondrial, nuclear, and microbial metagenomic compartments, as well as the historical development and current practice of genetic diagnostics and gene therapies targeting each compartment.
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15

Shelby, Elena-Silvia, Florina Mihaela Nedelea, Tanser Huseyinoglu, Relu Cocos, Mihaela Badina, Corina Sporea, Liliana Padure, and Andrada Mirea. "Innovative therapies in genetic diseases: Cystic fibrosis." Romanian Journal of Pediatrics 70, no. 1 (March 31, 2021): 16–20. http://dx.doi.org/10.37897/rjp.2021.1.3.

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Cystic fibrosis, also named mucoviscidosis, is the most frequent hereditary pulmonary disease and is produced by mutations in the CFTR gene, encoding an anionic channel for chloride and bicarbonate involved in the regulation of salt and bicarbonate metabolisms. Currently, about half of the patients with cystic fibrosis can benefit personalized therapy consisting in modulators, drugs which restore or improve the functionality and stability of CFTR. Moreover, presently, other therapies, such as gene therapy using the CRISP/CAS-9, modified antisense oligonucleotides or the insertion of the wild-type gene using nanolipidic particles or viral vectors, are being developed. This article aims to take stock of the principal types of cystic fibrosis therapies which have been approved or are in clinical trials.
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16

Wolfe, J. H. "Gene Therapy in Large Animal Models of Human Genetic Diseases." ILAR Journal 50, no. 2 (January 1, 2009): 107–11. http://dx.doi.org/10.1093/ilar.50.2.107.

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17

Lian, Jane B., and Christopher Niyibizi. "Breakout Session 4: Gene Therapy for Genetic Diseases of Bone." Clinical Orthopaedics and Related Research 379 (October 2000): S159—S163. http://dx.doi.org/10.1097/00003086-200010001-00021.

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18

Nerkar, A. G., and G. S. Chakraborthy. "Gene therapy." Current Trends in Pharmacy and Pharmaceutical Chemistry 3, no. 3 (July 15, 2021): 15–18. http://dx.doi.org/10.18231/j.ctppc.2021.005.

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Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease. Gene therapy is the introduction, removal or change in genetic material specifically DNA or RNA into the cells of a patient to treat a specific disease. Disease is caused to mutation. Genes influence all from the color of our hair to immune system, but genes are not built correctly. Targets of the gene therapy causing diseases. Gene therapy uses section of DNACurrently gene therapy is being tested only for disease that have no. other cures. Interest area of this review covers introduction, history, DNA transfer, Types of gene therapy, working of therapy, techniques, Delivery system, strategies, technique challenges, risks, gene introduction site and clinical applications of gene therapy.
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19

Candotti, Fabio. "Advances of gene therapy for primary immunodeficiencies." F1000Research 5 (March 9, 2016): 310. http://dx.doi.org/10.12688/f1000research.7512.1.

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In the recent past, the gene therapy field has witnessed a remarkable series of successes, many of which have involved primary immunodeficiency diseases, such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease, and Wiskott-Aldrich syndrome. While such progress has widened the choice of therapeutic options in some specific cases of primary immunodeficiency, much remains to be done to extend the geographical availability of such an advanced approach and to increase the number of diseases that can be targeted. At the same time, emerging technologies are stimulating intensive investigations that may lead to the application of precise genetic editing as the next form of gene therapy for these and other human genetic diseases.
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20

Fischer, Alain, Salima Hacein-Bey-Abina, and Marina Cavazzana-Calvo. "Gene therapy of metabolic diseases." Journal of Inherited Metabolic Disease 29, no. 2-3 (April 2006): 409–12. http://dx.doi.org/10.1007/s10545-006-0270-7.

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21

Liu, Yunbo, Xu Zhang, and Lin Yang. "Genetic Engineering of AAV Capsid Gene for Gene Therapy Application." Current Gene Therapy 20, no. 5 (December 11, 2020): 321–32. http://dx.doi.org/10.2174/1566523220666200930105521.

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Adeno-associated virus (AAV) is a promising vector for in vivo gene therapy because of its excellent safety profile and ability to mediate stable gene expression in human subjects. However, there are still numerous challenges that need to be resolved before this gene delivery vehicle is used in clinical applications, such as the inability of AAV to effectively target specific tissues, preexisting neutralizing antibodies in human populations, and a limited AAV packaging capacity. Over the past two decades, much genetic modification work has been performed with the AAV capsid gene, resulting in a large number of variants with modified characteristics, rendering AAV a versatile vector for more efficient gene therapy applications for different genetic diseases.
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22

Foreman, John. "Genetic Diseases of the Kidney." Open Urology & Nephrology Journal 8, no. 1 (November 26, 2015): 136–47. http://dx.doi.org/10.2174/1874303x015080100136.

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The number of genes associated with renal disease is increasing every day and this has led to a clearer understanding of the pathophysiology of renal disease in many disorders. It is also appreciated now that a genetic mutation(s) underlie many renal syndromes. Genetic testing may also offer the possibility to diagnose some renal diseases without the need for a renal biopsy. It also allows the prenatal diagnosis of certain renal diseases in at risk fetuses or identification of potential renal disease before it has become manifest. Finally, identification of a specific gene mutation holds the possibility of correction though gene therapy in the future. It is increasingly clear that many renal disorders in pediatrics are a consequence of genetic mutations. In the future, genetic testing will become as easy and as common as ordering a serum creatinine today.
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23

Sarin, Apurva. "Gene therapy in inflammatory diseases." Journal of Genetics 79, no. 2 (August 2000): 81. http://dx.doi.org/10.1007/bf02728950.

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24

Waddington, Simon N., Suzanne M. K. Buckley, and Charles Coutelle. "Intervention in utero: Fetal gene therapy." Biochemist 30, no. 3 (June 1, 2008): 7–9. http://dx.doi.org/10.1042/bio03003007.

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Postnatal gene therapy is a reactive approach to disease treatment. The disease in question will have been diagnosed, usually after symptoms have appeared. Children with lethal immune deficiency diseases have been treated by gene therapy with a high success rate in Paris and London, and many other clinical gene therapy trials for various genetic diseases, acquired conditions and different forms of cancer are presently underway or planned in adult patients.
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25

Bastola, Prabhakar, Liujiang Song, Brian C. Gilger, and Matthew L. Hirsch. "Adeno-Associated Virus Mediated Gene Therapy for Corneal Diseases." Pharmaceutics 12, no. 8 (August 13, 2020): 767. http://dx.doi.org/10.3390/pharmaceutics12080767.

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According to the World Health Organization, corneal diseases are the fourth leading cause of blindness worldwide accounting for 5.1% of all ocular deficiencies. Current therapies for corneal diseases, which include eye drops, oral medications, corrective surgeries, and corneal transplantation are largely inadequate, have undesirable side effects including blindness, and can require life-long applications. Adeno-associated virus (AAV) mediated gene therapy is an optimistic strategy that involves the delivery of genetic material to target human diseases through gene augmentation, gene deletion, and/or gene editing. With two therapies already approved by the United States Food and Drug Administration and 200 ongoing clinical trials, recombinant AAV (rAAV) has emerged as the in vivo viral vector-of-choice to deliver genetic material to target human diseases. Likewise, the relative ease of applications through targeted delivery and its compartmental nature makes the cornea an enticing tissue for AAV mediated gene therapy applications. This current review seeks to summarize the development of AAV gene therapy, highlight preclinical efficacy studies, and discuss potential applications and challenges of this technology for targeting corneal diseases.
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26

Yamada, André Katayama, Rozangela Verlengia, and Carlos Roberto Bueno Junior. "Myostatin: genetic variants, therapy and gene doping." Brazilian Journal of Pharmaceutical Sciences 48, no. 3 (September 2012): 369–77. http://dx.doi.org/10.1590/s1984-82502012000300003.

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Since its discovery, myostatin (MSTN) has been at the forefront of muscle therapy research because intrinsic mutations or inhibition of this protein, by either pharmacological or genetic means, result in muscle hypertrophy and hyperplasia. In addition to muscle growth, MSTN inhibition potentially disturbs connective tissue, leads to strength modulation, facilitates myoblast transplantation, promotes tissue regeneration, induces adipose tissue thermogenesis and increases muscle oxidative phenotype. It is also known that current advances in gene therapy have an impact on sports because of the illicit use of such methods. However, the adverse effects of these methods, their impact on athletic performance in humans and the means of detecting gene doping are as yet unknown. The aim of the present review is to discuss biosynthesis, genetic variants, pharmacological/genetic manipulation, doping and athletic performance in relation to the MSTN pathway. As will be concluded from the manuscript, MSTN emerges as a promising molecule for combating muscle wasting diseases and for triggering wide-ranging discussion in view of its possible use in gene doping.
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27

Wu, Shao-Shuai, Qing-Cui Li, Chang-Qing Yin, Wen Xue, and Chun-Qing Song. "Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases." Theranostics 10, no. 10 (2020): 4374–82. http://dx.doi.org/10.7150/thno.43360.

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28

Tamirisa, Kamala, and Debabrata Mukherjee. "Gene Therapy in Cardiovascular Diseases." Current Gene Therapy 2, no. 4 (December 1, 2002): 427–35. http://dx.doi.org/10.2174/1566523023347643.

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29

Gabriel, Rodney, and Guo-Yuan Yang. "Gene Therapy in Cerebrovascular Diseases." Current Gene Therapy 7, no. 6 (December 1, 2007): 421–33. http://dx.doi.org/10.2174/156652307782793496.

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30

Fumoto, Shintaro, Junya Nishi, Junzo Nakamura, and Koyo Nishida. "Gene Therapy for Gastric Diseases." Current Gene Therapy 8, no. 3 (June 1, 2008): 187–200. http://dx.doi.org/10.2174/156652308784746431.

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31

Chuang, Ya-Hui, Yao-Hsu Yang, Si-Jie Wu, and Bor-Luen Chiang. "Gene Therapy for Allergic Diseases." Current Gene Therapy 9, no. 3 (June 1, 2009): 185–91. http://dx.doi.org/10.2174/156652309788488604.

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32

Gonçalves, Giulliana Augusta Rangel, and Raquel de Melo Alves Paiva. "Gene therapy: advances, challenges and perspectives." Einstein (São Paulo) 15, no. 3 (September 2017): 369–75. http://dx.doi.org/10.1590/s1679-45082017rb4024.

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ABSTRACT The ability to make site-specific modifications to the human genome has been an objective in medicine since the recognition of the gene as the basic unit of heredity. Thus, gene therapy is understood as the ability of genetic improvement through the correction of altered (mutated) genes or site-specific modifications that target therapeutic treatment. This therapy became possible through the advances of genetics and bioengineering that enabled manipulating vectors for delivery of extrachromosomal material to target cells. One of the main focuses of this technique is the optimization of delivery vehicles (vectors) that are mostly plasmids, nanostructured or viruses. The viruses are more often investigated due to their excellence of invading cells and inserting their genetic material. However, there is great concern regarding exacerbated immune responses and genome manipulation, especially in germ line cells. In vivo studies in in somatic cell showed satisfactory results with approved protocols in clinical trials. These trials have been conducted in the United States, Europe, Australia and China. Recent biotechnological advances, such as induced pluripotent stem cells in patients with liver diseases, chimeric antigen receptor T-cell immunotherapy, and genomic editing by CRISPR/Cas9, are addressed in this review.
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33

Johnson, N. W., and M. A. Curtis. "Preventive Therapy for Periodontal Diseases." Advances in Dental Research 8, no. 2 (July 1994): 337–48. http://dx.doi.org/10.1177/08959374940080023001.

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Rational approaches to the prevention of destructive periodontitis should be based on a clear understanding of etiology and pathogenesis. However, we are dealing with a heterogeneous family of diseases in which different factors operate. It is an oversimplification to regard poor oral hygiene, and hence an accumulation of non-specific dental bacterial plaque, as the major risk factor. Epidemiological evidence indicates that host factors are likely to be of overriding importance for the most severe forms. The limitations of non-specific plaque control are therefore discussed. Specific inhibitors of virulence factors provide a logical approach, but their clinical application awaits improved knowledge. Improvement of general health and resistance to disease by proper nutrition, the avoidance of intercurrent disease, and elimination of smoking and stress-induced risk are encouraged. The genetic basis of susceptibility to periodontitis is increasingly understood, and, while gene therapy is not likely to be a practicable approach to prevention, genetic markers of risk are emerging.
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34

Lander, Eric S., and Harvey Lodish. "Mitochondrial diseases: Gene mapping and gene therapy." Cell 61, no. 6 (June 1990): 925–26. http://dx.doi.org/10.1016/0092-8674(90)90055-j.

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35

Bañuls, Lucía, Daniel Pellicer, Silvia Castillo, María Mercedes Navarro-García, María Magallón, Cruz González, and Francisco Dasí. "Gene Therapy in Rare Respiratory Diseases: What Have We Learned So Far?" Journal of Clinical Medicine 9, no. 8 (August 8, 2020): 2577. http://dx.doi.org/10.3390/jcm9082577.

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Gene therapy is an alternative therapy in many respiratory diseases with genetic origin and currently without curative treatment. After five decades of progress, many different vectors and gene editing tools for genetic engineering are now available. However, we are still a long way from achieving a safe and efficient approach to gene therapy application in clinical practice. Here, we review three of the most common rare respiratory conditions—cystic fibrosis (CF), alpha-1 antitrypsin deficiency (AATD), and primary ciliary dyskinesia (PCD)—alongside attempts to develop genetic treatment for these diseases. Since the 1990s, gene augmentation therapy has been applied in multiple clinical trials targeting CF and AATD, especially using adeno-associated viral vectors, resulting in a good safety profile but with low efficacy in protein expression. Other strategies, such as non-viral vectors and more recently gene editing tools, have also been used to address these diseases in pre-clinical studies. The first gene therapy approach in PCD was in 2009 when a lentiviral transduction was performed to restore gene expression in vitro; since then, transcription activator-like effector nucleases (TALEN) technology has also been applied in primary cell culture. Gene therapy is an encouraging alternative treatment for these respiratory diseases; however, more research is needed to ensure treatment safety and efficacy.
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36

SPENCER, REBECCA N., DAVID J. CARR, and ANNA L. DAVID. "GENE THERAPY FOR OBSTETRIC CONDITIONS." Fetal and Maternal Medicine Review 25, no. 3-4 (November 2014): 147–77. http://dx.doi.org/10.1017/s0965539515000030.

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The first clinical trials of gene therapy in the 1990s offered the promise of a new paradigm for the treatment of genetic diseases. Over the decades that followed the challenges and setbacks which gene therapy faced often overshadowed any successes. Despite this, recent years have seen cause for renewed optimism. In 2012 Glybera™, an adeno-associated viral vector expressing lipoprotein lipase, became the first gene therapy product to receive marketing authorisation in Europe, with a licence to treat familial lipoprotein lipase deficiency. This followed the earlier licensing in China of two gene therapies: Gendicine™ for head and neck squamous cell carcinoma and Oncorine™ for late-stage nasopharyngeal cancer. By this stage over 1800 clinical trials had been, or were being, conducted worldwide, and the therapeutic targets had expanded far beyond purely genetic disorders. So far no trials of gene therapy have been carried out in pregnancy, but an increasing understanding of the molecular mechanisms underlying obstetric diseases means that it is likely to have a role to play in the future. This review will discuss how gene therapy works, its potential application in obstetric conditions and the risks and limitations associated with its use in this setting. It will also address the ethical and regulatory issues that will be faced by any potential clinical trial of gene therapy during pregnancy.
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37

Factor, Phillip. "Gene Therapy for Acute Diseases." Molecular Therapy 4, no. 6 (December 2001): 515–24. http://dx.doi.org/10.1006/mthe.2001.0504.

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38

EVANS, C. H., J. D. WHALEN, C. H. EVANS, S. C. GHIVIZZANI, and P. D. ROBBINS. "Gene therapy in autoimmune diseases." Annals of the Rheumatic Diseases 57, no. 3 (March 1, 1998): 125–27. http://dx.doi.org/10.1136/ard.57.3.125.

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39

SCHNEIDER, M. "Gene therapy in inflammatory diseases." Annals of the Rheumatic Diseases 60, no. 2 (February 1, 2001): 105. http://dx.doi.org/10.1136/ard.60.2.105.

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40

Jhuma, Khadija Akther, ASM Giasuddin, AM Mujibul Haq, and Shahryar Nabi. "Gene therapy for primary immunode!ciency diseases: where are we now?" Bangladesh Medical Journal 44, no. 3 (April 17, 2016): 160–64. http://dx.doi.org/10.3329/bmj.v44i3.27377.

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In 1950s, double-stranded deoxyribonucleic acid (DNA) had been identified as the biochemical basis of heredity and ‘gene’ had been shown to be a segment of DNA. Accordingly, vast majority of hereditary disorders involve changes, i.e. mutations in specific genes. Can medical treatment leading to genetic cure of these hereditary disorders possible? The answer is ‘yes’ as the initial thoughts of ‘Gene therapy’ have been transformed into reality in recent times. Gene therapy is the application of various technologies including ‘Recombinant DNA Technology’ for introduction of a relevant functional gene, i.e. exogenous DNA, into a cell to achieve therapeutic effects for genetic disorders. Since 1990, gene therapy has become standard treatment for a number of primary immunodeficiency diseases (PIDs) such as adenosine deaminase deficiency form of severe combined immunodeficiency (ADA-SCID), SCID-X1, Wiskott-Aldrich syndrome (WAS), chronic granulomatous disease (CGD) and others. As we eagerly wait until the results of ongoing further clinical trials are available, updated accounts of the status of ‘Gene Therapy’ for selective PIDs are presented in this review article.Bangladesh Med J. 2015 Sep; 44 (3): 160-164
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41

Kay, M. A., and S. L. C. Woo. "Gene Therapy for Metabolic Diseases." ILAR Journal 36, no. 3-4 (January 1, 1994): 47–53. http://dx.doi.org/10.1093/ilar.36.3-4.47.

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42

Ohashi, Toya. "Gene therapy for lysosomal storage diseases and peroxisomal diseases." Journal of Human Genetics 64, no. 2 (November 29, 2018): 139–43. http://dx.doi.org/10.1038/s10038-018-0537-5.

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43

Fay, Alex J., Renatta Knox, Erin E. Neil, and Jonathan Strober. "Targeted Treatments for Inherited Neuromuscular Diseases of Childhood." Seminars in Neurology 40, no. 03 (April 15, 2020): 335–41. http://dx.doi.org/10.1055/s-0040-1702940.

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AbstractIn the past decade, the number of genes linked to neuromuscular diseases of childhood has expanded dramatically, and this genetic information is forming the basis for gene-specific and even mutation-specific therapies. At the forefront of these advances are the two recently approved treatments for spinal muscular atrophy: one, an antisense oligonucleotide that modifies splicing of the SMN2 gene, and, the other, a gene therapy vector that delivers the SMN1 gene to motor neurons, both of which are allowing patients to acquire developmental milestones previously unseen in this fatal disease. This review highlights these advances and emerging targeted therapies for Duchenne muscular dystrophy and centronuclear myopathy, while also covering enzyme replacement therapy and small molecule-based targeted therapies for conditions such as Pompe's disease and congenital myasthenic syndromes. With these and other newer techniques for targeted correction of genetic defects, such as CRISPR/Cas9, there is now hope that treatments for many more genetic diseases of the nervous system will follow in the near future.
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44

Kishnani, Priya S., Baodong Sun, and Dwight D. Koeberl. "Gene therapy for glycogen storage diseases." Human Molecular Genetics 28, R1 (June 22, 2019): R31—R41. http://dx.doi.org/10.1093/hmg/ddz133.

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AbstractThe focus of this review is the development of gene therapy for glycogen storage diseases (GSDs). GSD results from the deficiency of specific enzymes involved in the storage and retrieval of glucose in the body. Broadly, GSDs can be divided into types that affect liver or muscle or both tissues. For example, glucose-6-phosphatase (G6Pase) deficiency in GSD type Ia (GSD Ia) affects primarily the liver and kidney, while acid α-glucosidase (GAA) deficiency in GSD II causes primarily muscle disease. The lack of specific therapy for the GSDs has driven efforts to develop new therapies for these conditions. Gene therapy needs to replace deficient enzymes in target tissues, which has guided the planning of gene therapy experiments. Gene therapy with adeno-associated virus (AAV) vectors has demonstrated appropriate tropism for target tissues, including the liver, heart and skeletal muscle in animal models for GSD. AAV vectors transduced liver and kidney in GSD Ia and striated muscle in GSD II mice to replace the deficient enzyme in each disease. Gene therapy has been advanced to early phase clinical trials for the replacement of G6Pase in GSD Ia and GAA in GSD II (Pompe disease). Other GSDs have been treated in proof-of-concept studies, including GSD III, IV and V. The future of gene therapy appears promising for the GSDs, promising to provide more efficacious therapy for these disorders in the foreseeable future.
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45

MacLaren, Robert E. "Gene Therapy for Retinal Disease: What Lies Ahead." Ophthalmologica 234, no. 1 (2015): 1–5. http://dx.doi.org/10.1159/000438872.

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Gene therapy in simple terms can be defined as a medical treatment that exerts its effects using molecules of DNA or RNA within cells. Most traditional drugs act by mechanisms that include binding to cell surface receptors, inhibiting enzymes in intracellular pathways or by modifying transcription. These approaches rely to some extent on a normal genetic make-up of the cell in the final common pathway, which raises significant challenges in diseases that are caused by specific gene mutations. An alternative gene therapy approach to change the behaviour of cells at the most fundamental level by one single genetic modification is therefore potentially very powerful and wide ranging. This paper presents an overview of retinal gene therapy at the current time and highlights the future therapeutic potential for a number of diseases that are currently incurable.
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Saitoh, Youichi, Amami Kato, Yasushi Hagihara, Yasufumi Kaneda, and Toshiki Yoshimine. "Gene Therapy for Ischemic Brain Diseases." Current Gene Therapy 3, no. 1 (February 1, 2003): 49–58. http://dx.doi.org/10.2174/1566523033347561.

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47

Guo, Xiao-Lu, Tsai-Hua Chung, Yue Qin, Jie Zheng, Huyong Zheng, Liyuan Sheng, Tung Wynn, and Lung-Ji Chang. "Hemophilia Gene Therapy: New Development from Bench to Bed Side." Current Gene Therapy 19, no. 4 (November 18, 2019): 264–73. http://dx.doi.org/10.2174/1566523219666190924121836.

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Novel gene therapy strategies have changed the prognosis of many inherited diseases in recent years. New development in genetic tools and study models has brought us closer to a complete cure for hemophilia. This review will address the latest gene therapy research in hemophilia A and B including gene therapy tools, genetic strategies and animal models. It also summarizes the results of recent clinical trials. Potential solutions are discussed regarding the current barriers in gene therapy for hemophilia.
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48

Garlick, J. A., and E. S. Fenjves. "Keratinocyte Gene Transfer and Gene Therapy." Critical Reviews in Oral Biology & Medicine 7, no. 3 (July 1996): 204–21. http://dx.doi.org/10.1177/10454411960070030101.

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Gene therapy has moved beyond the pre-clinical stage to the treatment of a variety of inherited and acquired diseases. For such therapy to be successful, genes must be efficiently delivered to target cells and gene products must be expressed for prolonged periods of time without toxic effects to the host. This may be achieved by means of an in vivo strategy where genes are transferred directly into a host cell, or by means of an ex vivo approach through which cells are removed, cultured, targeted for gene delivery, and grafted back to the host. Several obstacles continue to delay safe and effective clinical application of gene therapy in a variety of target cells. The limited survival of transplanted cells, transient expression of transferred genes, and difficulties in targeting stem cells are technical issues requiring further investigation. Epidermal and oral keratinocytes are potential vehicles for gene therapy. Several features of these tissues can be utilized to achieve delivery of therapeutic gene products for local or systemic delivery. These qualities include: (1) the presence of stem cells; (2) the cell-, strata-, and site-specific regulation of keratinocyte gene expression; (3) tissue accessibility; and (4) secretory capacity. Such features can be exploited by the use of gene therapy strategies to facilitate: (1) identification, enrichment, and targeting of stem cells to ensure the continued presence of the transferred gene; (2) high-level and persistent transgene expression using keratinocyte-specific promoters; (3) tissue access needed for culture and grafting for ex vivo therapy and direct in vivo gene transfer; (4) secretion of transgene product for local or systemic delivery; and (5) monitoring of genetically modified tissue and removal if treatment termination is required. Optimal gene therapy strategies are being tested in a variety of tissues to treat dominant and recessive genetic disorders as well as acquired diseases such as neoplasia and infectious disease. This experience provides a basis for the application of such clinical studies to a spectrum of diseases effecting epidermal and oral keratinocytes. Gene therapy is in an early stage yet holds great promise for its ultimate clinical application.
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Muldoon, Leslie L., and Edward A. Neuwelt. "Local and global gene therapy in the central nervous system." Behavioral and Brain Sciences 18, no. 1 (March 1995): 76–78. http://dx.doi.org/10.1017/s0140525x00037572.

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AbstractFor focal neurodegenerative diseases or brain tumors, localized delivery of protein or genetic vectors may be sufficient to alleviate symptoms, halt disease progression, or even cure the disease. One may circumvent the limitation imposed by the blood-brain barrier by transplantation of genetically altered cell grafts or focal inoculation of virus or protein. However, permanent gene replacement therapy for diseases affecting the entire brain will require global delivery of genetic vectors. The neurotoxicity of currently available viral vectors and the transient nature of transgene expression invivomust be overcome before their use in human gene therapy becomes clinically applicable.
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Sari, Bunga Anggreini, Azalia Talitha Zahra, Ganda Purba Tasti, and Ziske Maritska. "Healing the Fundamental Unit of Heredity (Gene Therapy): Current Perspective and What the Future Holds." Molecular and Cellular Biomedical Sciences 5, no. 2 (July 6, 2021): 62. http://dx.doi.org/10.21705/mcbs.v5i2.202.

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The ability to make precise adjustments to the human genome has been a goal of healing in which gene also introduces as the fundamental unit of heredity, in biomolecular technology in genetic diseases have opened new knowledge such as gene therapy. Gene therapy is a technique to repair DNA where its usage is to treat the malignancy and inherited genetic diseases. Gene therapy is a choice to the genetic cloth that goals to remedy a sickness this is hard to deal with or perhaps has no treatment. Currently, gene remedy is done in approaches to patients, specifically embryonic cells and somatic cells, every in vivo and ex vivo. Moral considerations with modification of the difficulty's cells and oversight of regulation and reagents want to be taken into consideration within the gene therapy project. Applications for using gene remedies have begun to be widely used, which include in case of maximum cancers, coronary heart disorder, infectious sicknesses, and others. Gene therapy has spread to a wide range of applications then go beyond the modification of genetic disorders. Advances in genetic modification of cancer cells and immunity and the use of viruses and bacteria to control cancer cells have resulted in many clinical trials and product developments for cancer treatment. The miracles and blessings of gene therapy are might believe, but even though they are being studied and developed now and, in the future, so that the desire for gene therapy may be even better future.Keywords: gene therapy, genetic recombination, gene therapy application
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