Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model

Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is an autosomal recessive motor neuron disease affecting children. It is caused by mutations in the IGHMBP2 gene (11q13) and presently has no cure. Recently, adeno-associated virus serotype 9 (AAV9)–mediated gene therapy has been show...

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Vydáno v:Sci Adv
Hlavní autoři: Nizzardo, Monica, Simone, Chiara, Rizzo, Federica, Salani, Sabrina, Dametti, Sara, Rinchetti, Paola, Del Bo, Roberto, Foust, Kevin, Kaspar, Brian K., Bresolin, Nereo, Comi, Giacomo P., Corti, Stefania
Médium: Artigo
Jazyk:Inglês
Vydáno: American Association for the Advancement of Science 2015
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Research Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4643829/
https://ncbi.nlm.nih.gov/pubmed/26601156
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.1500078
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