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    • Duchenne muscular dystrophy
    • Infant Spinal muscular atrophy
    • SMA-PME and Farber’s disease
    • Myotubular myopathy
    • Limb girdle muscular dystrophies
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Archives: Publication

Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation.

February 19, 2021

Exploiting the CRISPR/Cas9 system to study alternative splicing in vivo: application to titin.

February 19, 2021

In Vivo Gene Delivery to Lymph Node Stromal Cells Leads to Transgene-specific CD8+ T Cell Anergy in Mice.

February 19, 2021

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics.

February 19, 2021

Quantitative NMRI and NMRS identify augmented disease progression after loss of ambulation in forearms of boys with Duchenne muscular dystrophy.

February 19, 2021

Evaluation of the serum matrix metalloproteinase-9 as a biomarker for monitoring disease progression in Duchenne muscular dystrophy.

February 19, 2021

Systemic epigenetic response to recombinant lentiviral vectors independent of proviral integration

February 19, 2021

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.

February 19, 2021

Germline duplication of ATG2B and GSKIP predisposes to familial myeloid malignancies.

February 19, 2021

Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies.

February 19, 2021

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