Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent

Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is an autosomal recessive disease occurring during childhood. The gene responsible for disease development is a ubiquitously expressed protein, IGHMBP2. Mutations in IGHMBP2 result in the loss of α-motor neurons leading to muscle atro...

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Bibliografiska uppgifter
I publikationen:Mol Ther
Huvudupphovsmän: Shababi, Monir, Feng, Zhihua, Villalon, Eric, Sibigtroth, Christine M, Osman, Erkan Y, Miller, Madeline R, Williams-Simon, Patricka A, Lombardi, Abby, Sass, Thalia H, Atkinson, Arleigh K, Garcia, Michael L, Ko, Chien-Ping, Lorson, Christian L
Materialtyp: Artigo
Språk:Inglês
Publicerad: Nature Publishing Group 2016
Ämnen:
Original Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC4881770/
https://ncbi.nlm.nih.gov/pubmed/26860981
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.33
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