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      • Progressive muscular dystrophies
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    • Duchenne muscular dystrophy
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    • Myotubular myopathy
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    • Chronic septic granulomatosis
    • Severe Combined Immunodefiency (SCID)
    • Cori Forbes disease
    • GSD1a – Glygogen Storage Disease Type 1a
    • Crigler-Najjar syndrome
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Archives: Publication

Immunopathology in the brain of mice following vertical transmission of Coxsackievirus B4

June 30, 2021

SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson’s Disease

June 30, 2021

AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy

June 30, 2021

Combined Use of CFTR Correctors in LGMD2D Myotubes Improves Sarcoglycan Complex Recovery

June 30, 2021

Progress and challenges of gene therapy for Pompe disease.

February 19, 2021

Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle.

February 19, 2021

Balancing immunity and tolerance in gene therapy for inherited and acquired diseases.

February 19, 2021

Tailoring the CRISPR system to transactivate coagulation gene promoters in normal and mutated contexts.

February 19, 2021

Vectofusin-1 improves transduction of primary human cells with diverse retroviral and lentiviral pseudotypes, enabling robust, automated closed-system manufacturing.

February 19, 2021

In Vivo Monitoring of Calpain Activity by Forster Resonance Energy Transfer

February 19, 2021

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