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

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

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...

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Main Authors: Andreas C. Chai (Author), Francesco Chemello (Author), Hui Li (Author), Takahiko Nishiyama (Author), Kenian Chen (Author), Yu Zhang (Author), Efraín Sánchez-Ortiz (Author), Adeeb Alomar (Author), Lin Xu (Author), Ning Liu (Author), Rhonda Bassel-Duby (Author), Eric N. Olson (Author)
Format: Book
Published: Elsevier, 2023-06-01T00:00:00Z.
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