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Academic literature on the topic 'Motoer Neuron Disease – Therapy'

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Published: 4 June 2021
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Journal articles on the topic "Motoer Neuron Disease – Therapy"

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Sendtner, Michael. "Gene therapy for motor neuron disease." Nature Medicine 3, no. 4 (April 1997): 380–81. http://dx.doi.org/10.1038/nm0497-380.

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Kiernan, Matthew C. "Progress towards therapy in motor neuron disease." Nature Reviews Neurology 14, no. 2 (January 19, 2018): 65–66. http://dx.doi.org/10.1038/nrneurol.2017.186.

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&NA;. "Gene therapy helps mice with motor neuron disease." Inpharma Weekly &NA;, no. 1085 (May 1997): 10. http://dx.doi.org/10.2165/00128413-199710850-00026.

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Messina, Sonia. "New Directions for SMA Therapy." Journal of Clinical Medicine 7, no. 9 (August 31, 2018): 251. http://dx.doi.org/10.3390/jcm7090251.

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Spinal muscular atrophy (SMA) is a severe disorder of motor neurons and the most frequent genetic cause of mortality in childhood, due to respiratory complications. The disease occurs due to mutations in the survival motor neuron 1 (SMN1) gene that leads to a reduction in the SMN protein, causing degeneration of lower motor neurons, muscle weakness and atrophy. Recently, the Food and Drug Administration (FDA) and the European Medical Agency (EMA) approved the antisense oligonucleotide nusinersen, the first disease-modifying treatment for SMA. Encouraging results from SMN1 gene therapy studies have raised hope for other therapeutic approaches that might arise in the coming years. However, nusinersen licensing has created ethical, medical, and financial implications that will need to be addressed. In this review, the history and challenges of the new SMA therapeutic strategies are highlighted.
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Rosenfeld, Jeffrey, and Amy Ellis. "Nutrition and Dietary Supplements in Motor Neuron Disease." Physical Medicine and Rehabilitation Clinics of North America 19, no. 3 (August 2008): 573–89. http://dx.doi.org/10.1016/j.pmr.2008.03.001.

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Mackinnon, H. "Motor neuron disease and music therapy - a holistic approach." BMJ Supportive & Palliative Care 1, no. 2 (September 1, 2011): 204. http://dx.doi.org/10.1136/bmjspcare-2011-000100.16.

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Werner, Jana, Ilijas Jelcic, Esther Irene Schwarz, Elisabeth Probst-Müller, Jakob Nilsson, Bernhard Schwizer, Konrad Ernst Bloch, Andreas Lutterotti, Hans-Heinrich Jung, and Bettina Schreiner. "Anti-IgLON5 Disease." Neurology - Neuroimmunology Neuroinflammation 8, no. 2 (February 2, 2021): e962. http://dx.doi.org/10.1212/nxi.0000000000000962.

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ObjectiveTo expand the spectrum of anti-IgLON5 disease by adding 5 novel anti-IgLON5–seropositive cases with bulbar motor neuron disease-like phenotype.MethodsWe characterized the clinical course, brain MRI and laboratory findings, and therapy response in these 5 patients.ResultsPatients were severely affected by bulbar impairment and its respiratory consequences. Sleep-related breathing disorders and parasomnias were common. All patients showed clinical or electrophysiologic signs of motor neuron disease without fulfilling the diagnostic criteria for amyotrophic lateral sclerosis. One patient regained autonomy in swallowing and eating, possibly related to immunotherapy.ConclusionIgLON5 disease is an important differential diagnosis to evaluate in patients with bulbar motor neuron disease-like phenotype and sleep disorders. There is need for a deeper understanding of the underlying pathobiology to determine whether IgLON5 disease is an immunotherapy-responsive condition.
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Manzano, Raquel, Gianni Sorarú, Christopher Grunseich, Pietro Fratta, Emanuela Zuccaro, Maria Pennuto, and Carlo Rinaldi. "Beyond motor neurons: expanding the clinical spectrum in Kennedy’s disease." Journal of Neurology, Neurosurgery & Psychiatry 89, no. 8 (January 20, 2018): 808–12. http://dx.doi.org/10.1136/jnnp-2017-316961.

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Kennedy’s disease, or spinal and bulbar muscular atrophy (SBMA), is an X-linked neuromuscular condition clinically characterised by weakness, atrophy and fasciculations of the limb and bulbar muscles, as a result of lower motor neuron degeneration. The disease is caused by an abnormally expanded triplet repeat expansions in the ubiquitously expressed androgen receptor gene, through mechanisms which are not entirely elucidated. Over the years studies from both humans and animal models have highlighted the involvement of cell populations other than motor neurons in SBMA, widening the disease phenotype. The most compelling aspect of these findings is their potential for therapeutic impact: muscle, for example, which is primarily affected in the disease, has been recently shown to represent a valid alternative target for therapy to motor neurons. In this review, we discuss the emerging study of the extra-motor neuron involvement in SBMA, which, besides increasingly pointing towards a multidisciplinary approach for affected patients, deepens our understanding of the pathogenic mechanisms and holds potential for providing new therapeutic targets for this disease.
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Villalón, E., R. A. Kline, C. E. Smith, Z. C. Lorson, E. Y. Osman, S. O’Day, L. M. Murray, and C. L. Lorson. "AAV9-Stathmin1 gene delivery improves disease phenotype in an intermediate mouse model of spinal muscular atrophy." Human Molecular Genetics 28, no. 22 (July 31, 2019): 3742–54. http://dx.doi.org/10.1093/hmg/ddz188.

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Abstract Spinal muscular atrophy (SMA) is a devastating infantile genetic disorder caused by the loss of survival motor neuron (SMN) protein that leads to premature death due to loss of motor neurons and muscle atrophy. The approval of an antisense oligonucleotide therapy for SMA was an important milestone in SMA research; however, effective next-generation therapeutics will likely require combinatorial SMN-dependent therapeutics and SMN-independent disease modifiers. A recent cross-disease transcriptomic analysis identified Stathmin-1 (STMN1), a tubulin-depolymerizing protein, as a potential disease modifier across different motor neuron diseases, including SMA. Here, we investigated whether viral-based delivery of STMN1 decreased disease severity in a well-characterized SMA mouse model. Intracerebroventricular delivery of scAAV9-STMN1 in SMA mice at P2 significantly increased survival and weight gain compared to untreated SMA mice without elevating Smn levels. scAAV9-STMN1 improved important hallmarks of disease, including motor function, NMJ pathology and motor neuron cell preservation. Furthermore, scAAV9-STMN1 treatment restored microtubule networks and tubulin expression without affecting tubulin stability. Our results show that scAAV9-STMN1 treatment improves SMA pathology possibly by increasing microtubule turnover leading to restored levels of stable microtubules. Overall, these data demonstrate that STMN1 can significantly reduce the SMA phenotype independent of restoring SMN protein and highlight the importance of developing SMN-independent therapeutics for the treatment of SMA.
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Francis, Kathleen, John R. Bach, and Joel A. DeLisa. "Evaluation and rehabilitation of patients with adult motor neuron disease." Archives of Physical Medicine and Rehabilitation 80, no. 8 (August 1999): 951–63. http://dx.doi.org/10.1016/s0003-9993(99)90089-8.

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Dissertations / Theses on the topic "Motoer Neuron Disease – Therapy"

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Zuardi, Marina Campos. "Quantificação da lesão neuronal e mielínica na Esclerose Lateral Amiotrófica através da ressonância magnética." Universidade de São Paulo, 2012. http://www.teses.usp.br/teses/disponiveis/17/17140/tde-30082012-083717/.

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Introdução: A Esclerose Lateral Amiotrófica (ELA) é uma doença degenerativa e progressiva que afeta neurônios motores da medula espinhal, tronco cerebral e/ ou córtex motor. Sua manifestação clínica é bastante variada, sua etiologia desconhecida e a progressão, fatal. Não existe ainda um tratamento curativo para a ELA, porém alguns medicamentos e a realização de fisioterapia podem auxiliar, fornecendo ao paciente uma melhor qualidade de vida. Objetivos: Testar a hipótese de que técnicas quantitativas de Ressonância Magnética (RM) são eficazes para detectar a lesão neuronal no encéfalo de pacientes com ELA no estágio inicial da doença e, verificar se existe correlação entre a lesão encefálica e a perda funcional do paciente. Dessa forma, pretende-se estabelecer um protocolo capaz de contribuir para o diagnóstico precoce da ELA. Metodologia: Quinze pacientes com diagnóstico de ELA definida ou provável (12 homens e três mulheres), com idade entre 37 e 79 anos e seus respectivos controles foram submetidos a um protocolo de avaliação por RM, que incluiu um estudo estrutural volumétrico e quantitativo do dano neuronal e mielínico por razão de sequências ponderadas em T1 e FLAIR, da Transferência de Magnetização (MT), Relaxometria, Anisotropia Fracionada (FA) e Difusão (DTI), além da Espectroscopia de prótons. Alguns dos sujeitos foram submetidos também à uma avaliação física de força muscular, de funcionalidade através da Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R), de qualidade de vida através da Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) e de quantificação da fadiga pela Fadigue Severity Scale (FSS). Os dados da RM dos dois grupos foram comparados através da análise de variância multi e univariada e submetidos à correção de múltiplas comparações de Bonferroni. Nas variáveis com diferença significante entre os grupos, foi estudada a validade preditiva da medida, calculando-se a área sob a curva ROC e estabelecidos os valores de sensibilidade, especificidade e acurácia. Os dados das escalas foram comparados pelo teste de Mann-Whitney e correlacionados entre si e com as estruturas através da correlação de Spearman. Resultados: As várias técnicas da RM, com exceção da MT, identificaram pelo menos uma estrutura com diferença significante entre os dois grupos, totalizando 11 estruturas mais a razão dos metabólitos NAA/Cre. Os valores de sensibilidade, especificidade e acurácia foram satisfatórios variando entre 0,60 e 1,00 , com destaque para o Volume do Giro Superior Frontal Direito e Giro Superior Frontal Esquerdo que apresentaram valores 1,00 , 0,93 e 0,97 , respectivamente. As escalas ALSFRS-R e ALSAQ-40 apresentaram diferença significativa entre os dois grupos, mas a FSS não apresentou. As escalas apresentaram correlação significativa entre si em quase todos os escores totais e domínios. Já a correlação das escalas com as estruturas foi significativa apenas para o Volume. Conclusões: Técnicas como DTI, FA, Relaxometria e Volume se mostraram mais eficazes no diagnóstico precoce de pacientes com ELA do que as outras. A redução de volume de substância cinzenta se correlacionou positivamente com a ALSFRS-R. Por fim, propomos um protocolo para avaliação de pacientes com ELA, que inclua imagem volumétrica de alta resolução para cálculo da Volumetria e DTI.
Introduction: Amyotrophic Lateral Sclerosis (ALS) is a progressive and degenerative disease that affects motor neurons in the spinal cord, brain stem and/ or motor cortex. Their clinical presentation is varied, its unknown etiology and fatal progression. There isnt still a curative treatment for ALS, but some medications and physical therapy can help by providing the patient a better quality of life. Objectives: To test the hypothesis that quantitative techniques of magnetic resonance imaging (MRI) are effective to detect neuronal damage in the brain of patients with ALS at the inicial stage of the disease and see if there is a correlation between brain injury and functional loss of the patient. Thus, we intend to establish a protocol can to contribute to early diagnosis of ALS. Methods: Fifteen patients with definite or probable ALS (12 men and three women) aged between 37 and 79 and their respective controls underwent an MRI evaluation protocol, including a volumetric and quantitative structural study of damage neuronal and myelin by reason of T1-weighted sequences and FLAIR , Magnetization Transfer (MT), Relaxometry, Fractional Anisotropy (FA), Diffusion (DTI) and Proton magnetic resonance spectroscopy. Some of the subjects also underwent a physical assessment of muscle strength, functionality by Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R), quality of life through the Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) and quantification of fatigue by Fadigue Severity Scale (FSS). The MRI data of the two groups were compared using analysis of variance multivariate and univariate and submitted to correction for multiple comparisons of Bonferroni. In the variables with significant differences between groups, we studied the predictive validity of the measure, by calculating the area under the ROC curve and set the sensitivity, specificity and accuracy. The data of the scales were compared using Mann-Whitney test and correlated with each other and with the structures by Spearman correlation. Results: The various MRI techniques, with the exception of MT, identified at least one structure with a significant difference between the two groups, a total of 11 structures over the reason for the metabolites NAA/Cre. The sensitivity, specificity and accuracy were satisfactory ranging from 0.60 to 1.00 , with emphasis on Volume of Gyros Superior Frontal Right and Gyros Superior Frontal Left that averaged 1.00 , 0.93 and 0,97 , respectively. The ALSFRS-R and ALSAQ-40 scales showed significant differences between the two groups, but the FSS did not. The scales were significantly correlated with each other in almost all domains and total scores. The correlation with the structures of the scales was significant only for the Volume. Conclusions: Techniques such as DTI, FA, Relaxometry and Volume are more effective in early diagnosis of ALS patients than others. The decrease in gray matter volume was positively correlated with the ALSFRS-R. Finally, we propose a protocol for evaluation patients with ALS, including high-resolution volumetric image to calculate the Volume and DTI.
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Eleftheriadou, Ioanna. "Development of novel targeted lentiviral vectors for gene therapy of motor neuron diseases." Thesis, Imperial College London, 2013. http://hdl.handle.net/10044/1/27243.

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Motor Neuron Diseases (MND) including Amyotrophic Lateral Sclerosis (ALS) and Spinal Muscular Atrophy (SMA) are neurodegenerative diseases that can cause progressive paralysis and premature death, while there are no treatments available up to date. Gene therapy using lentiviruses has been successful at alleviating symptoms and extending survival in murine models of both. Restricting transduction to specific cells, is critical for safe and efficient gene therapy. The aim of this study was to generate novel lentiviral vectors with tropism to motor neurons (MNs) using surface engineering, that involves incorporation of a fusogenic glycoprotein (mutated sindbis G) and an antibody recognizing a cell-surface receptor, onto the lentiviral surface. Antibodies against rat Thy1.1, rat p75NTR (Low Affinity Nerve Growth Factor Receptor) and mouse CAR (Coxsackie and adenovirus receptor) receptors expressed on the presynaptic terminal of the neuromuscular junction (NMJ) were cloned and used to surface engineer high titer lentiviral vectors ( Thy1.1, p75NTR and CAR). These vectors preferentially transduced cell lines expressing these receptors and primary motor neuron cultures compared to non-targeted controls. The ability of these vectors to be transported retrogradely and transduce MNs was demonstrated in vitro in compartmented microfluidic cultures in fixed and live imaging experiments. Tropism of these vectors was assessed in vivo in the rat brain following intrastiatal injections. In vivo intramuscular delivery of CAR targeted lentiviral vectors in tibialis anterior of mice lead to transduction of motor neurons in ventral spinal cord. Transduction of spinal cord was further demonstrated by in vivo bioluminescence imaging studies in mice injected intramuscularly with the CAR targeted vectors. This is the first ever demonstration that surface engineering can confer novel trafficking and transduction characteristics to lentiviral vectors. These targeted lentiviral vectors have superior trafficking, transduction and specificity for MNs than previously used lentiviral vectors, making them good candidates for non-invasive CNS -targeted delivery of therapeutics in MN diseases.
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Islam, Tarin A. "Characterisation of axonal retrograde transport of rabies pseudotyped lentiviral vectors for application in gene therapy of motor neuron diseases." Thesis, Imperial College London, 2013. http://hdl.handle.net/10044/1/25142.

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Lentiviral vectors, such as those derived from Human Immunodeficiency Virus-1 (HIV-1) and Equine Infectious Anaemia Virus (EIAV), can be targeted to the neurons by replacing their natural envelope with rabies-G glycoprotein (RV-G), through a process known as pseudotyping. They are retrogradedly transported to distal projecting neurons in vivo where transgene expression occurs, an approach with significant potential for gene therapy of motor neuron diseases. However, the molecular processes that underlie retrograde transduction are unchartered and barrier(s) which result in low transduction efficiencies are thus not defined. The project aimed to characterise the processes involved in the entry and endocytic trafficking of RV-G pseudotyped lentiviral vectors in a differentiated motor neuron cell line, NSC-34, and primary rat motor neurons. For the first time, the project demonstrates the co-internalisation of RV-G pseudotyped lentiviral vectors with its 3 specific receptors, namely p75 neurotrophin receptor (p75NTR), neural cell adhesion molecule (NCAM) and nicotinic acetylcholine receptor (nAchR). Furthermore, internalised lentiviral vectors follow a sequential Rab5 to Rab7 endosomal maturation along the axonal endocytic pathway. Using the process of tetracysteine tagging, a small genetic tag was successfully introduced into the matrix of the vector capsid and labelling of the tag with the biarsenical dye FlAsH was optimised. Utilising these and other differently labelled vectors, we demonstrate through live cell imaging, that it is the RV-G pseudotype that confers on the vectors the ability to traffic retrogradedly. This trafficking pathway utilises non-acidic axonal carriers as previously demonstrated for the p75NTR and tetanus toxin trafficking in motor neurons. Additionally, the nAchR is also targeted to this retrograde trafficking pathway. Furthermore retrograde transport was studied in compartmented microfluidic motor neuron cultures and this trafficking was found to be both rapid and efficient. This suggests that the barrier(s) to efficient transduction with these vectors is post axonal transport. Additionally, studies to isolate and characterise axonal retrograde endosomes in primary motor neurons based on a system of pull-down of magnetic nano particles (MNPs) bound to a streptavidin displaying lentiviral vector were performed. The project optimised streptavidin expressing vector production and shown their ability to bind biotin coated magnetic nano particles (MNPs) and transduce motor neurons with increased efficiency. Magnetic pull-down of the vector containing endosomes and subsequent proteomics have revealed a number of potential trafficking partners of RV-G pseudotyped lentiviral vectors. Taken together, this thesis characterises the processes involved in the entry and endocytic trafficking of RV-G pseudotyped lentiviral vectors and identifies the barrier to efficient transduction of motor neurons as post axonal transport and thereby opening up avenues to improve gene therapy vectors.
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Kennedy, Zachary C. "Optimizing CRISPR/Cas9 for Gene Silencing of SOD1 in Mouse Models of ALS." eScholarship@UMMS, 2019. https://escholarship.umassmed.edu/gsbs_diss/1047.

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Mutations in the SOD1 gene are the best characterized genetic cause of amyotrophic lateral sclerosis (ALS) and account for ~20% of inherited cases and 1-3% of sporadic cases. The gene-editing tool Cas9 can silence mutant genes that cause disease, but effective delivery of CRISPR-Cas9 to the central nervous system (CNS) remains challenging. Here, I developed strategies using canonical Streptococcus pyogenes Cas9 to silence SOD1. In the first strategy, I demonstrate effectiveness of systemic delivery of guide RNA targeting SOD1 to the CNS in a transgenic mouse model expressing human mutant SOD1 and Cas9. Silencing was observed in both the brain and the spinal cord. In the second strategy, I demonstrate the effectiveness of delivering both guide RNA and Cas9 via two AAVs into the ventricles of the brain of SOD1G93A mice. Silencing was observed in the brain and in motor neurons within the spinal cord. For both strategies, treated mice had prolonged survival when compared to controls. Treated mice also had improvements in grip strength and rotarod function. For ICV treated mice, we detected a benefit of SOD1 silencing using net axonal transport assays, a novel method to detect motor neuron function in mice before onset of motor symptoms. These studies demonstrate that Cas9-mediated genome editing can mediate disease gene silencing in motor neurons and warrants further development for use as a therapeutic intervention for SOD1-linked ALS patients.
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Mecca, Jordan. "Rôle des cellules souches musculaires dans la physiopathologie de l’amyotrophie spinale." Electronic Thesis or Diss., Sorbonne université, 2019. http://www.theses.fr/2019SORUS261.

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L’amyotrophie spinale (SMA), est une maladie neuromusculaire caractérisée par une dégénérescence des motoneurones (MN), une atrophie musculaire et une paralysie conduisant à une mort précoce dans les formes les plus sévères. La SMA est due à une réduction de la protéine ubiquitaire SMN résultant de mutations homozygotes dans le gène SMN1. Longtemps considérée comme une pathologie purement neuronale, la SMA apparaît aujourd’hui comme une pathologie multisystémique affectant de nombreux tissus périphériques, dont le muscle squelettique et les cellules souches musculaires (CS). Avec les premiers succès de la thérapie génique basée sur l’AAV9-SMN, émergent aussi des incertitudes quant aux effets à long terme de ces thérapies en particulier sur l’intégrité du système neuromusculaire. Ces travaux se sont inscrits dans cette problématique, et apportent un nouvel éclairage sur l’implication des CS musculaires dans la physiopathologie de la SMA. Nous avons observé une diminution du nombre de CS dans les muscles de patients SMA Type II, qui pourrait résulter d’un défaut d’engagement des CS vers la quiescence et d’une perte des CS quiescentes par apoptose. Grâce au modèle murin Pax7CreERT2/+;SmnF7/F7, nous avons démontré que cette déplétion des CS induite par le déficit en SMN induit, à long terme, une perte sélective des MN-α accompagnée de changements phénotypiques des fibres musculaires. Enfin, nous avons montré une dérégulation du profil d’expression des miARN dans les CS de souris SMA, et identifié de potentielles cibles thérapeutiques pour le développement de futures stratégies thérapeutiques combinées, restaurant SMN et préservant le système neuromusculaire sur le long terme
Spinal muscular atrophy (SMA) is a neuromuscular disorder characterized by motor neurons (MN) degeneration, muscle atrophy and paralysis leading to premature death in the most severe forms. SMA is due to a reduction of the ubiquitous protein called SMN resulting from homozygous mutations in SMN1 gene. Long considered as a purely neuronal disease, SMA appears now as a multisystemic disease affecting many peripheral tissues, including skeletal muscle and muscle stem cells (SC). With the first successes of AAV9-SMN-based gene therapy, uncertainties emerge about the long-term effects of these therapies, particularly regarding the integrity of the neuromuscular system. This work is in line with this problematic, and shed new light on the involvement of muscle SC in SMA pathophysiology. We observed a decreased number of SC in the muscles of SMA Type II patients, which could result from reduced ability of SMN-deficient SC to commit to quiescence and a loss of quiescent SC by apoptosis. Using the murine conditional KO model Pax7CreERT2/+;SmnF7/F7, we demonstrated that this SC-depletion induced by SMN deficiency leads, in the long term, to a selective loss of α-MN and phenotypic changes in muscle fibers. Finally, we showed a deregulation of miRNA expression profile in SMA mouse SC, and identified potential new therapeutic targets for the development of future combined therapeutic strategies, restoring SMN and preserving the neuromuscular system in the long term
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Vucic, Ostoja Steve School of Medicine UNSW. "The pathophysiology of amyotrophic lateral sclerosis." 2007. http://handle.unsw.edu.au/1959.4/40729.

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This thesis examines the pathophysiology of motor neurone dysfunction, along with site of disease onset, in amyotrophic lateral sclerosis (ALS). The rationale for this thesis is the "dying forward" hypothesis, which suggests that corticomotoneurons cause anterograde excitotoxic degeneration of motor neurons in ALS. Initially, axonal excitability studies were applied to ALS patients and revealed widespread axonal ion channel dysfunction, with increases in persistent Na+ conductances and reduction in K+ currents. Such changes result in axonal hyperexcitability, thereby resulting in generation of fasciculations and cramps. Subsequently, axonal excitability studies were applied to Kennedy's disease (KD) patients, a pathological control group, revealing similar changes to ALS and suggesting that upregulation of persistent Na+ conductances was responsible for generation of fasciculations. To better understand the mechanisms underlying fatigability and to assess whether Na+/K+ pump dysfunction contributes to neurodegeneration in ALS, activity-dependent changes in axonal excitability were measured after a maximal voluntary contraction. The increase in threshold was more pronounced in ALS patients with predominantly lower motor neuron involvement, suggesting that peripheral factors were responsible for fatigue in ALS and that Na+/K+ pump function was preserved. Having documented abnormalities of axonal excitability, a novel threshold tracking transcranial magnetic stimulation (TMS) technique was developed for assessment of cortical excitability. This technique overcomes the marked variability in the motor evoked potential with consecutive stimuli, a major limitation of the previous "constant stimulus" technique. After establishing normative data, threshold tracking TMS established that cortical hyperexcitability was an early and prominent feature in ALS. Similar changes were found in flail-arm variant ALS, a pure lower motor neuron form of ALS. In KD patients, cortical excitability was normal, thereby suggesting that cortical hyperexcitability is a primary event in ALS rather than a down-regulation of inhibitory control over the motor cortex in order to compensate for anterior horn cell loss. In order to determine whether cortical hyperexcitability underlies motor neurodegeneration, longitudinal studies were undertaken in familial ALS subjects with the copper/zinc superoxide-dismutase-1 gene mutation. These studies established that cortical hyperexcitability precedes the development of clinical ALS, thereby suggesting that cortical hyperexcitability underlies the basis of motor neurodegeneration in familial ALS.
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Lin, Chin-Lung, and 林金龍. "Monotherapy and Combination of electroacupuncture and mesenchymal stem cell therapy affects recovery of motor function and dopaminergic neuron degeneration in the mouse model of Parkinson’s disease." Thesis, 2018. http://ndltd.ncl.edu.tw/handle/xn4d5r.

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碩士
中國醫藥大學
中獸醫碩士學位學程
106
Neurodegenerative diseases are a serious problem worldwide, affecting neurons in the human brain and causing the deterioration or death of nerve cells, leading to movement disorders or physical and mental dysfunction. An example of a neurodegenerative disease is Parkinson''s disease (PD), which currently has no treatment to cure or prevent the progression of Parkinson''s disease. In this study, we used intraperitoneal injection (i.p.) of 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine, MPTP) to induce mouse Parkinson''s disease and explored the beneficial effects of electroacupuncture (EA) at Yanglingquan (GB34) and Taichong (LR3) acupoints, nasal administration of mesenchymal stem cells (MSCs) and their combination. In MPTP-treated mice of the Rotarod test, the latency of the EA group and the combination of the EA plus nasal administration of (MSCs) were significantly longer than that of MPTP only group. The latency of MSC group was no significant increased compared with the MPTP only group. The immunohistochemical analysis of tyrosine hydroxylase in the substantia nigra pars compacta (SNc) brain region of MPTP-treated mice showed that more TH-positive cells were observed in the (EA) group and the combination group compared with MPTP only group. There was no significant difference between the MSC group alone and the MPTP only group. The CD90+ CD44+ content of the isolated mesenchymal stem cells was tested by flow cytometry. The results showed that the CD90+ CD44+ in the isolated mesenchymal stem cells was about 8.92-14.10%. The above results show that EA treatment may be an effective strategy for patients with Parkinson''s disease. The low concentration of mesenchymal stem cells used in this study may be the reason why mesenchymal stem cells treatment has no curative effect.
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Books on the topic "Motoer Neuron Disease – Therapy"

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Motor neuron diseases: Causes, classification, and treatments. Hauppauge, N.Y: Nova Science Publishers, 2011.

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Spasticity: Diagnosis and management. New York: Demos Medical Pub., 2011.

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Rutherford, Kunel, and John L. R. Forsythe. Motor Neuron Disease. Saunders Ltd., 2002.

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W, Kuncl Ralph, ed. Motor neuron disease. London: W.B. Saunders, 2002.

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L, Mancini Raffaele, ed. Motor neuron disease research progress. New York: Nova Biomedical Books, 2008.

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J, Strong Michael, ed. Dementia and motor neuron disease. Abingdon [England]: Informa UK Ltd., 2006.

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(Editor), David Oliver, Gian Domenico Borasio (Editor), and Declan Walsh (Editor), eds. Palliative Care in Amyotrophic Lateral Sclerosis (Motor Neuron Disease). Oxford University Press, USA, 2000.

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(Editor), Michael P. Barnes, and Garth R. Johnson (Editor), eds. Upper Motor Neurone Syndrome and Spasticity: Clinical Management and Neurophysiology. Cambridge University Press, 2001.

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1952-, Barnes Michael P., and Johnson Garth R. 1945-, eds. Upper motor neurone syndrome and spasticity: Clinical management and neurophysiology. New York, NY: Cambridge University Press, 2001.

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Eisen, Andrew. Motor neurone disease. Oxford University Press, 2015. http://dx.doi.org/10.1093/med/9780199658602.003.0009.

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In this chapter, the following ten key events in motor neurone disease, also known as amyotrophic lateral sclerosis (ALS), are considered: the first description of ALS by Cruveilhier; discovery of the first SOD1 mutation; use of the ALSFRS (functional rating scale) for determining therapeutic trial outcomes; the contentious issue of establishing the site of onset of ALS; clinical, pathological, and molecular evidence indicating that frontotemporal dementia and ALS are closely related; demonstration that ALS bears some resemblance to the transmissible spongiform encephalopathies; use of Riluzole as the approved therapy for ALS; the major inflammatory component of ALS; a Guamanian disorder that is biochemically and ultrastructurally similar to that of Alzheimer’s disease; and awareness that the true onset of ALS is unknown but certainly precedes clinical onset by years or decades.
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Book chapters on the topic "Motoer Neuron Disease – Therapy"

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Towne, Chris, and Patrick Aebischer. "Lentiviral and Adeno-Associated Vector-Based Therapy for Motor Neuron Disease Through RNAi." In Methods in Molecular Biology™, 87–108. Totowa, NJ: Humana Press, 2009. http://dx.doi.org/10.1007/978-1-60327-295-7_7.

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Tysnes, Ole-Bjørn. "Motor Neuron Disease (Amyotrophic Lateral Sclerosis)." In Handbook of Neurological Therapy, 165–71. Oxford University Press, 2014. http://dx.doi.org/10.1093/med/9780199862924.003.0017.

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Sakowski, Stacey A., J. Simon Lunn, and Eva L. Feldman. "Stem Cell Therapy for Motor Neuron Disease." In Motor Neuron Disease in Adults, 312–28. Oxford University Press, 2014. http://dx.doi.org/10.1093/med/9780199783113.003.0033.

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Sharma, Alok, Hemangi Sane, Nandini Gokulchandran, Prerna Badhe, Amruta Paranjape, Radhika Pradhan, Rohit Das, and Hema Biju. "Stem Cell Therapy in Motor Neuron Disease." In Novel Aspects on Motor Neuron Disease. IntechOpen, 2020. http://dx.doi.org/10.5772/intechopen.87116.

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"Nutrition in neurological conditions." In Oxford Handbook of Nutrition and Dietetics, edited by Joan Webster-Gandy, Angela Madden, and Michelle Holdsworth, 781–94. Oxford University Press, 2020. http://dx.doi.org/10.1093/med/9780198800132.003.0033.

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Takeda, Akitoshi, and Bruce Miller. "Frontotemporal dementias." In New Oxford Textbook of Psychiatry, edited by John R. Geddes, Nancy C. Andreasen, and Guy M. Goodwin, 405–13. Oxford University Press, 2020. http://dx.doi.org/10.1093/med/9780198713005.003.0041.

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Fronto-temporal dementia (FTD) is a heterogenous clinical syndrome with a progressive decline in behaviour, executive function, and language. Approximately 40% of FTD patients report a family history, and 10–15% of FTD cases show an autosomal dominant pattern of inheritance. FTD often mimics psychiatric disorders because of the prominent behavioural features, and particularly in the early stages, the only manifestation of illness may be purely behavioural. There is significant clinical, pathological, and genetic overlap between FTD and motor neuron disease/amyotrophic lateral sclerosis (FTD-ALS) and two atypical Parkinsonian syndromes—progressive supranuclear palsy (PSP) and cortico-basal degeneration (CBD). FTD is associated with non-Alzheimer’s disease pathology and the molecular aggregation of specific proteins—tau, TAR-DNA binding protein (TDP), and fused in sarcoma (FUS). Advances in clinical, imaging, and molecular characterization have increased the accuracy of FTD diagnosis. At present, appropriate management of FTD symptoms involves a combination of pharmacological therapy with techniques involving behavioural, physical, and environmental manipulation.
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Donsante, A. "Gene Therapy for Amyotrophic Lateral Sclerosis." In Molecular and Cellular Therapies for Motor Neuron Diseases, 167–205. Elsevier, 2017. http://dx.doi.org/10.1016/b978-0-12-802257-3.00008-0.

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Miller, M. R., E. Y. Osman, and C. L. Lorson. "Gene Therapy for Spinal Muscular Atrophy." In Molecular and Cellular Therapies for Motor Neuron Diseases, 233–50. Elsevier, 2017. http://dx.doi.org/10.1016/b978-0-12-802257-3.00010-9.

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Faravelli, I., and S. Corti. "Cellular Therapy for Spinal Muscular Atrophy." In Molecular and Cellular Therapies for Motor Neuron Diseases, 251–75. Elsevier, 2017. http://dx.doi.org/10.1016/b978-0-12-802257-3.00011-0.

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O'Connor, D. M. "Introduction to Gene and Stem-Cell Therapy." In Molecular and Cellular Therapies for Motor Neuron Diseases, 141–65. Elsevier, 2017. http://dx.doi.org/10.1016/b978-0-12-802257-3.00007-9.

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Conference papers on the topic "Motoer Neuron Disease – Therapy"

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Stockdale, Claire, Richard Price, Trish Campbell, and Lisa Sievwright. "136 A review of the use of mucolytic agents in Motor Neurone Disease (MND). Is there benefit to using multiple agents over mono-therapy?" In Accepted Oral and Poster Abstract Submissions, The Palliative Care Congress 1 Specialty: 3 Settings – home, hospice, hospital 25 – 26 March 2021 | A virtual event, hosted by Make it Edinburgh Live, the Edinburgh International Conference Centre’s hybrid event platform. British Medical Journal Publishing Group, 2021. http://dx.doi.org/10.1136/spcare-2021-pcc.154.

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