Výsledky vyhledávání - "Mary J. Wetz"

Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy

Autor: Mary J. Wetz, Christian L. Lorson, Monir Shababi, Megan M. Krogman, Jacqueline J. Glascock

Publikováno v: Biochemical and biophysical research communications. 417(1)

Spinal Muscular Atrophy (SMA), an autosomal recessive neuromuscular disorder, is the leading genetic cause of infant mortality. SMA is caused by the homozygous loss of Survival Motor Neuron-1 (SMN1). SMA, however, is not due to complete absence of SM

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7cbd5b31c9979f85593dfce951f023bb
https://pubmed.ncbi.nlm.nih.gov/22172949

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Decreasing Disease Severity in Symptomatic, Smn−/−;SMN2+/+, Spinal Muscular Atrophy Mice Following scAAV9-SMN Delivery

Autor: Christian L. Lorson, Megan M. Krogman, Mary J. Wetz, Erkan Y. Osman, Jacqueline J. Glascock, Monir Shababi

Spinal muscular atrophy (SMA), an autosomal recessive neuromuscular disorder, is the leading genetic cause of infant mortality. SMA is caused by the homozygous loss of Survival Motor Neuron-1 (SMN1). In humans, a nearly identical copy gene is present

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::64e8ad53627f8a1da4049f1979953579
https://europepmc.org/articles/PMC3300100/

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