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Archives: Publication

Perspectives on best practices for gene therapy programs.

February 19, 2021

Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs.

February 19, 2021

Ultrasound assessment of the diaphragm: Preliminary study of a canine model of X-linked myotubular myopathy.

February 19, 2021

Genomic analysis of sleeping beauty transposon integration in human somatic cells.

February 19, 2021

Mouse models of primary central nervous system lymphomas: tools for basing funding and therapeutic strategies.

February 19, 2021

The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer.

February 19, 2021

Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-beta (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis.

February 19, 2021

Proteomics profiling of urine reveals specific titin fragments as biomarkers of Duchenne muscular dystrophy.

February 19, 2021

Phosphoinositide substrates of myotubularin affect voltage-activated Ca(2)(+) release in skeletal muscle.

February 19, 2021

Blockade of ActRIIB signaling triggers muscle fatigability and metabolic myopathy.

February 19, 2021

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