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

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

Abstract 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 thera...

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Bibliographic Details
Main Authors: Michelle A. Farrar, Loudella Calotes-Castillo, Ranil De Silva, Peter Barclay, Lani Attwood, Julie Cini, Monica Ferrie, Didu S. Kariyawasam
Format: Article
Language:English
Published: SpringerOpen 2023-11-01
Series:Molecular and Cellular Pediatrics
Subjects:
Spinal muscular atrophy
Newborn screening
Gene therapy
Rare diseases
Clinical translation
Implementation
Online Access:https://doi.org/10.1186/s40348-023-00171-5

Internet

https://doi.org/10.1186/s40348-023-00171-5

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