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A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1

Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is an infantile autosomal recessive disease caused by the loss of the ubiquitously expressed IGHMBP2 gene. SMARD1 causes degeneration of alpha-motor neurons, resulting in distal muscle weakness, diaphragm paralysis, and respiratory ma...

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Detalles Bibliográficos
Autores principales:	Shababi, Monir, Villalón, Eric, Kaifer, Kevin A., DeMarco, Vince, Lorson, Christian L.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6127875/ https://www.ncbi.nlm.nih.gov/pubmed/30202772 http://dx.doi.org/10.1016/j.omtm.2018.08.005

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