Promising therapeutic approaches using CRISPR/Cas9 genome editing technology in the treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy is an X-linked recessive hereditary monogenic disorder caused by inability to produce dystrophin protein. In most patients, the expression of dystrophin lost due to disrupting mutations in open reading frame. Despite the efforts in a large number of different therapeutic...
Main Authors: | , , , , |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2021-03-01
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Series: | Genes and Diseases |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2352304220300015 |