Gene modification strategies using AO-mediated exon skipping and CRISPR/Cas9 as potential therapies for Duchenne muscular dystrophy patients
Duchenne muscular dystrophy (DMD) is an X-linked genetic disease affecting 1 in 5000 young males worldwide annually. Patients experience muscle weakness and loss of ambulation at an early age, with ∼75% reduced life expectancy. Recently developed genetic editing strategies aim to convert severe DMD...
Main Authors: | , , |
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Format: | Article |
Language: | English |
Published: |
Wiley
2020-12-01
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Series: | Engineering Biology |
Subjects: | |
Online Access: | https://digital-library.theiet.org/content/journals/10.1049/enb.2020.0017 |