Medicina Molecular e Genética

CRISPR/Cas9 and its Delivery Methods for Duchenne Muscular Dystrophy (DMD) Gene Therapies

Abstract

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 efficiency of gene editing is the delivery vector. Lot of attempts has been made to create an efficient therapy for DMD with CRISPR/Cas9, but still, the major hurdles rely on delivery techniques. Therefore, optimization of the delivery methods will support the complete regenerative therapy for DMD in future. This review mainly concentrates on the various aspects of CRISPR/Cas9 technologies and its delivery methods used in developing therapies for DMD and its optimization possibilities.