TY  - JOUR
AU  - Dara, Mahintaj
AU  - Dianatpour, Mehdi
AU  - Azarpira, Negar
AU  - Tanideh, Nader
PY  - 2025
DA  - 2025/01/08
TI  - Gene Therapy Strategies for Muscular Dystrophies: Current Insights and Future Directions
JO  - OBM Genetics
SP  - 280
VL  - 09
IS  - 01
AB  - Gene therapy, a groundbreaking method for addressing genetic mutations, includes strategies such as gene repair, replacement, inactivation, or the introduction of therapeutic genes, circumventing traditional surgical or pharmacological approaches. Delivery through viral or non-viral vectors presents trade-offs in efficiency and immune response. Recent gene-editing technologies like ZFNs, TALENs, and CRISPR facilitate precise genome modifications by inducing targeted double-strand breaks, with CRISPR/Cas9 recognized for its versatility. Muscular dystrophies, marked by progressive muscle degeneration due to genetic mutations, are a significant focus for gene therapy. While a definitive cure remains elusive, gene therapy provides hope, with ongoing research investigating tailored approaches for various types of muscular dystrophy. This review highlights gene therapy's potential in treating muscular dystrophies, concentrating on the diverse strategies under exploration and contributing to the quest for effective therapeutic interventions and, potentially, cures for these debilitating conditions.
SN  - 2577-5790
UR  - https://doi.org/10.21926/obm.genet.2501280
DO  - 10.21926/obm.genet.2501280
ID  - Dara2025
ER  -