Gene Therapy of Muscle Diseases

Gene therapy uses genes to treat or prevent diseases such as muscle diseases. Here are the latest discoveries pertaining to gene therapy and muscle diseases.

May 30, 2020

Successful Gene Delivery to Cardiac Allograft with Adeno-Associated Viral Vector Using Ex Vivo Storage Perfusion Platform

The Journal of Heart and Lung Transplantation : the Official Publication of the International Society for Heart Transplantation
Y P ChiangC A Milano
April 29, 2020
Open Access

Novel CAPN3 variant associated with an autosomal dominant calpainopathy

Neuropathology and Applied Neurobiology
Mathieu CerinoShahram Attarian
May 1, 2020
Review
Open Access

Noncoding RNAs in Duchenne and Becker muscular dystrophies: role in pathogenesis and future prognostic and therapeutic perspectives

Cellular and Molecular Life Sciences : CMLS
Roberta BrusaStefania Corti
July 2, 2020

Moving forward after two deaths in a gene therapy trial of myotubular myopathy

Human Gene Therapy
James M Wilson, Terence R Flotte
May 1, 2020

RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model

Molecular Therapy : the Journal of the American Society of Gene Therapy
Osasumwen V AimiuwuWayne N Frankel
May 17, 2020
Open Access

Mitochondrial Dysfunction: A Common Hallmark Underlying Comorbidity between sIBM and Other Degenerative and Age-Related Diseases

Journal of Clinical Medicine
Marc Catalán-GarcíaJosep M Grau-Junyent
June 12, 2020

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

Molecular Therapy : the Journal of the American Society of Gene Therapy
Umut CaginFederico Mingozzi
April 29, 2020
Review

Gene therapy for haemophilia

The Cochrane Database of Systematic Reviews
Akshay SharmaUlrike M Reiss
May 12, 2020
Open Access

Blood-derived biomarkers correlate with clinical progression in Duchenne muscular dystrophy

Journal of Neuromuscular Diseases
Kristin StrandbergCristina Al-Khalili Szigyarto
June 17, 2020
Review
Open Access

Molecular therapies in childhood neuromuscular disorders-definite hope versus unknown pitfalls

Bundesgesundheitsblatt, Gesundheitsforschung, Gesundheitsschutz
Astrid BlaschekUlrike Schara
June 27, 2020
Review
Open Access

Klotho and the Treatment of Human Malignancies

Cancers
Aishani SachdevaIrene Chong
May 30, 2020

AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice

Molecular Therapy : the Journal of the American Society of Gene Therapy
Aurore BesseMaria Grazia Biferi
May 25, 2020
Review

BMP gene delivery for skeletal tissue regeneration

Bone
Maxim BezDan Gazit
May 2, 2020
Open Access

Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?

Molecular Therapy. Methods & Clinical Development
Karim BeyMarie-Anne Colle

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