Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2(nmd-2J)

Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is an autosomal recessive disorder that develops in infancy and arises from mutation of the immunoglobulin helicase μ-binding protein 2 (IGHMBP2) gene. Whereas IGHMBP2 is ubiquitously expressed, loss or reduction of function leads to...

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Detalles Bibliográficos
Autores principales: Shababi, Monir, Smith, Caley E., Ricardez Hernandez, Sara M., Marquez, Jose, Al Rawi, Zayd, Villalón, Eric, Farris, K. David, Garro-Kacher, Mona O., Lorson, Christian L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8426477/
https://www.ncbi.nlm.nih.gov/pubmed/34553000
http://dx.doi.org/10.1016/j.omtm.2021.07.008

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