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Archives: Publication

Peripheral Cellular Immune Responses Induced by Subretinal Adeno-Associated Virus Gene Transfer Can Be Restrained by the Subretinal-Associated Immune Inhibition Mechanism

August 19, 2024

Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice

August 19, 2024

Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy

August 19, 2024

Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver

August 19, 2024

SETDB1 modulates the TGFβ response in Duchenne muscular dystrophy myotubes

August 19, 2024

A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III

August 19, 2024

Exome sequencing in undiagnosed congenital myopathy reveals new genes and refines genes–phenotypes correlations

August 19, 2024

An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II

August 19, 2024

Generation of iPSC lines from three Laing distal myopathy patients with a recurrent MYH7 p.Lys1617del variant

August 19, 2024

Generation of two iPSC lines from adult central core disease patients with dominant missense variants in the RYR1 gene

August 19, 2024

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