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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...
Autores principales: | Sierra-Delgado, Julieth Andrea, Sinha-Ray, Shrestha, Kaleem, Abuzar, Ganjibakhsh, Meysam, Parvate, Mohini, Powers, Samantha, Zhang, Xiaojin, Likhite, Shibi, Meyer, Kathrin |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2023
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Materias: | |
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 |
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