Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective

Onasemnogene abeparvovec has been life-changing for children with spinal muscular atrophy (SMA), signifying the potential and progress occurring in gene- and cell-based therapies for rare genetic diseases. Hence, it is important that clinicians gain knowledge and understanding in gene therapy-based...

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Detalles Bibliográficos
Autores principales: Farrar, Michelle A., Calotes-Castillo, Loudella, De Silva, Ranil, Barclay, Peter, Attwood, Lani, Cini, Julie, Ferrie, Monica, Kariyawasam, Didu S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10645685/
https://www.ncbi.nlm.nih.gov/pubmed/37964159
http://dx.doi.org/10.1186/s40348-023-00171-5

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