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.

April 17, 2021
Open Access

Patient-specific iPSC-derived cellular models of LGMDR1.

Stem Cell Research
A J Mateos-AierdiA López de Munain
April 16, 2021

Human antigen R promotes lung fibroblast differentiation to myofibroblasts and increases extracellular matrix production.

Journal of Cellular Physiology
Fatmah Al-HabeebCarolyn J Baglole
April 16, 2021
Review
Open Access

Blepharospasm, Oromandibular Dystonia, and Meige Syndrome: Clinical and Genetic Update.

Frontiers in Neurology
Hongying MaQiang Qu
April 14, 2021
Open Access

Base editing repairs an SGCA mutation in human primary muscle stem cells.

JCI Insight
Helena EscobarSimone Spuler
April 12, 2021

Histological Analysis of Tibialis Anterior Muscle of DMDmdx4Cv Mice from 1 to 24 Months.

Journal of Neuromuscular Diseases
Sabrina Ben LarbiBénédicte Chazaud
April 12, 2021
Open Access

Costs and health resource use in patients with X-linked myotubular myopathy: insights from U.S. commercial claims.

Journal of Managed Care & Specialty Pharmacy
Naomi C SacksRobert J Graham
April 12, 2021

Potential targets identified in adenoid cystic carcinoma point out new directions for further research.

American Journal of Translational Research
Zhenan LiuTingting Yu
April 10, 2021
Review
Open Access

Viral gene therapy for paediatric neurological diseases: progress to clinical reality.

Developmental Medicine and Child Neurology
Riccardo PrivolizziJoanne Ng
April 8, 2021
Open Access

Helicobacter canis bacteraemia in a rheumatoid arthritis patient treated with tofacitinib: case report and literature review.

Annals of Clinical Microbiology and Antimicrobials
Matic MihevcIztok Holc
April 5, 2021
Review
Open Access

Anti-AAV Antibodies in AAV Gene Therapy: Current Challenges and Possible Solutions.

Frontiers in Immunology
Thomas Weber
April 4, 2021
Review

New advances in Amyotrophic Lateral Sclerosis genetics: Towards gene therapy opportunities for familial and young cases.

Revue neurologique
M-D-M AmadorS Millecamps
April 4, 2021
Open Access

Nutraceutical Screening in a Zebrafish Model of Muscular Dystrophy: Gingerol as a Possible Food Aid.

Nutrients
Rosario LicitraFilippo M Santorelli
April 3, 2021
Open Access

SLCO1B1 Phenotype and CYP3A5 Polymorphism Significantly Affect Atorvastatin Bioavailability.

Journal of Personalized Medicine
Pablo ZubiaurFrancisco Abad-Santos
April 3, 2021
Open Access

Muscle Proteomic Profile before and after Enzyme Replacement Therapy in Late-Onset Pompe Disease.

International Journal of Molecular Sciences
Manuela MoriggiCecilia Gelfi
April 3, 2021
Review
Open Access

Myotonic dystrophy type 1 drug development: a pipeline toward the market.

Drug Discovery Today
Marta Pascual-GilabertRuben Artero

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