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Review Article
CRISPR/Cas9 and its Delivery Methods for Duchenne Muscular Dystrophy
(DMD) Gene Therapies
Author(s): Shalini T*
Duchenne Muscular Dystrophy (DMD) is a X linked genetic disorder that causes difficulty in walking, followed
by progressive skeletal muscle degeneration and some cardiac muscle related issues, that threatens the life
expectancy of patients. This condition is due a mutation in a gene that produces cytoskeletal protein termed as
dystrophin. Targeting this gene to correct or bypass the mutation would benefit in effective therapies for DMD.
CRISPR/Cas9 (Clustered Regularly Interspaced Palindromic Repeats) technology has created an evolution in
precise gene modification techniques. With the help of a guide RNA, Cas9 (a DNA endonuclease) can create a
double strand breaks to carry out the targeted gene modifications. By simply modifying the guide RNA sequences,
Cas9 can be used for flexible programming of new target sites. The prime factor that determines the therapeutic.. Read More»
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