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

• Main Authors: Michelle A. Farrar (Author), Loudella Calotes-Castillo (Author), Ranil De Silva (Author), Peter Barclay (Author), Lani Attwood (Author), Julie Cini (Author), Monica Ferrie (Author), Didu S. Kariyawasam (Author)
• Format: Book
• Published: SpringerOpen, 2023-11-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...