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

Internet

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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