Single-swap editing for the correction of common Duchenne muscular dystrophy mutations

• Автори: Andreas C. Chai (Автор), Francesco Chemello (Автор), Hui Li (Автор), Takahiko Nishiyama (Автор), Kenian Chen (Автор), Yu Zhang (Автор), Efraín Sánchez-Ortiz (Автор), Adeeb Alomar (Автор), Lin Xu (Автор), Ning Liu (Автор), Rhonda Bassel-Duby (Автор), Eric N. Olson (Автор)
• Формат: Книга
• Опубліковано: Elsevier, 2023-06-01T00:00:00Z.
• Предмети: article
• Онлайн доступ: Connect to this object online.

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive disease of progressive muscle weakness and wasting caused by the absence of dystrophin protein. Current gene therapy approaches using antisense oligonucleotides require lifelong dosing and have limited efficacy in restoring dystrophin p...