In Vitro Modeling as a Tool for Testing Therapeutics for Spinal Muscular Atrophy and IGHMBP2-Related Disorders

SIMPLE SUMMARY: Spinal Muscular Atrophy (SMA) is a genetic disease that can cause infant mortality. It is typically caused by mutations in the SMN1 gene. On the other hand, mutations in the IGHMBP2 gene can lead to a range of diseases, including the rare form of SMA known as SMARD1, as well as Charc...

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Detalles Bibliográficos
Autores principales: Sierra-Delgado, Julieth Andrea, Sinha-Ray, Shrestha, Kaleem, Abuzar, Ganjibakhsh, Meysam, Parvate, Mohini, Powers, Samantha, Zhang, Xiaojin, Likhite, Shibi, Meyer, Kathrin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10295315/
https://www.ncbi.nlm.nih.gov/pubmed/37372153
http://dx.doi.org/10.3390/biology12060867

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10295315/
https://www.ncbi.nlm.nih.gov/pubmed/37372153
http://dx.doi.org/10.3390/biology12060867

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