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      • Progressive muscular dystrophies
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    • Duchenne muscular dystrophy
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    • Fanconi’s anemia
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    • Cori Forbes disease
    • GSD1a – Glygogen Storage Disease Type 1a
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Archives: Publication

Ataxia with oculomotor apraxia type 2: A clinical and genetic study of 19 patients

February 19, 2021

An Association Study of 22 Candidate Genes in Psoriasis Families Reveals Shared Genetic Factors with Other Autoimmune and Skin Disorders

February 19, 2021

Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models

February 19, 2021

An Autosomal-Recessive Form of Cutis Laxa Is Due to Homozygous Elastin Mutations, and the Phenotype May Be Modified by a Heterozygous Fibulin 5 Polymorphism

February 19, 2021

Insertional transformation of hematopoietic cells by selfinactivating lentiviral and gammaretroviral vectors

February 19, 2021

Calcium-dependent plasma membrane repair requires m- or mu-calpain, but not calpain-3, the proteasome, or caspases

February 19, 2021

Diverse genomic integration of a lentiviral vector developed for the treatment of Wiskott-Aldrich syndrome

February 19, 2021

RNA-targeting approaches for neuromuscular diseases

February 19, 2021

A comparison of synthetic oligodeoxynucleotides, DNA fragments and AAV-1 for targeted episomal and chromosomal gene repair

February 19, 2021

Cardiac ankyrin repeat protein is a marker of skeletal muscle pathological remodelling

February 19, 2021

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