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.

July 28, 2020
Open Access

A Mitochondrial tRNA Mutation Causes Axonal CMT in a Large Venezuelan Family

Annals of Neurology
Alexander FayLouis J Ptáček
August 8, 2020
Open Access

DOK7 Gene Therapy Enhances Neuromuscular Junction Innervation and Motor Function in Aged Mice

IScience
Ryo UetaYuji Yamanashi
August 14, 2020
Review
Open Access

Electroporation-Based Treatments in Urology

Cancers
Aleksander KiełbikJulita Kulbacka
July 17, 2020
Open Access

MicroRNA-19a Targets Fibroblast Growth Factor-Inducible Molecule 14 and Prevents Tubular Damage in Septic AKI

Analytical Cellular Pathology (Amsterdam)
Jun HongShi-Jing Mo
September 15, 2020
Open Access

S100A4 Silencing Facilitates Corneal Wound Healing After Alkali Burns by Promoting Autophagy via Blocking the PI3K/Akt/mTOR Signaling Pathway

Investigative Ophthalmology & Visual Science
Yulin WangQiong Zhou
September 6, 2020

Engineering carotenoid production in mammalian cells for nutritionally enhanced cell-cultured foods

Metabolic Engineering
Andrew J StoutDavid L Kaplan
August 21, 2020
Open Access

Novel heterozygous truncating titin variants affecting the A-band are associated with cardiomyopathy and myopathy/muscular dystrophy

Molecular Genetics & Genomic Medicine
Kelly A RichJennifer Roggenbuck
August 11, 2020
Case Report
Open Access

Apolipoprotein AI-Derived Vitreous Amyloidosis: An Elusive Diagnosis

Case Reports in Ophthalmology
Juan B YepezJ Fernando Arevalo
August 14, 2020
Review
Open Access

Hydrogel-Based Localized Nonviral Gene Delivery in Regenerative Medicine Approaches-An Overview

Pharmaceutics
Natalia Carballo-PedraresAna Rey-Rico
August 11, 2020
Review
Open Access

Effects of RAGE inhibition on the progression of the disease in hSOD1G93A ALS mice

Pharmacology Research & Perspectives
Liping LiuMariana Pehar
August 19, 2020
Open Access

Cowden syndrome is a risk factor for multiple neoplasm: a case report

World Journal of Surgical Oncology
Sofia MigueloteJ P Araújo Teixeira
August 21, 2020
Open Access

Ovine congenital progressive muscular dystrophy (OCPMD) is a model of TNNT1 congenital myopathy

Acta Neuropathologica Communications
Joshua S ClaytonKristen J Nowak
September 5, 2020
Review

Overview of Gene Therapy in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy

Pediatric Pulmonology
Nicolas J Abreu, Megan A Waldrop
September 9, 2020

Cationic amphiphilic drugs as potential anti-cancer therapy for bladder cancer

Molecular Oncology
Geertje van der HorstEllen C Zwarthoff
July 23, 2020
Open Access

Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy

Molecular Therapy. Methods & Clinical Development
Virginie MariotJulie Dumonceaux
September 5, 2020
Review

Phosphorus dendrimers as powerful nanoplatforms for drug delivery, as fluorescent probes and for liposome interaction studies: A concise overview

European Journal of Medicinal Chemistry
Dzmitry ShcharbinJean-Pierre Majoral
September 5, 2020
Open Access

Founder Mutation in N-terminus of Cardiac Troponin I Causes Malignant Hypertrophic Cardiomyopathy

Circulation. Genomic and Precision Medicine
Akl C FahedChristine E Seidman

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