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    • Duchenne muscular dystrophy
    • Infant Spinal muscular atrophy
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Archives: Publication

Improvement of synthetic vectors for gene therapy using ring-opening cationic polymerization

February 19, 2021

Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy

February 19, 2021

Safety and Efficacy of AAV-Mediated Calpain 3 Gene Transfer in a Mouse Model of Limb-Girdle Muscular Dystrophy Type 2A

February 19, 2021

Dynamic reprogramming of DNA methylation at an epigenetically sensitive allele in mice

February 19, 2021

Synthesis, characterization, and gene transfer application of poly(ethylene glycol-b-ethylenimine) with high molar mass polyamine block.

February 19, 2021

Linear topology confers in vivo gene transfer activity to polyethylenimines

February 19, 2021

Efficient control of gene expression in the hematopoietic system using a single Tet-on inducible lentiviral vector

February 19, 2021

A mice model for monitoring calpain activity in physiological and pathological conditions

February 19, 2021

Calpain-3 mutations in Turkey

February 19, 2021

Spine deformities in Charcot-Marie-Tooth 4C caused by SH3TC2 gene mutations

February 19, 2021

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