Safety and efficacy of AAV-based mini- and micro-dystrophin gene therapies in Duchenne muscular dystrophy: a systematic review and meta-analysis of clinical trials

Haya Nassour, Samer A Al_Shbailat, Arwa Sobhi Ibrahim, Amal Mohammad Allan, Mostafa Saad Badie Aboudeeb, Qais M Alnjoom, Mohammad Jebril Farhan Alrfou, George Elian Makhoul, Mahmoud Ghassan Weis, Rasha K. Ahmad, Sara M. Abdelstar

Open source

DOI
10.1136/jmg-2026-111649
Published
2026-09-16
Container
Journal of Medical Genetics
Publisher
BMJ
Open access
unknown

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BibTeX

@article{allodium:10.1136/jmg-2026-111649,
  title = {Safety and efficacy of AAV-based mini- and micro-dystrophin gene therapies in Duchenne muscular dystrophy: a systematic review and meta-analysis of clinical trials},
  author = {Haya Nassour and Samer A Al\_Shbailat and Arwa Sobhi Ibrahim and Amal Mohammad Allan and Mostafa Saad Badie Aboudeeb and Qais M Alnjoom and Mohammad Jebril Farhan Alrfou and George Elian Makhoul and Mahmoud Ghassan Weis and Rasha K. Ahmad and Sara M. Abdelstar},
  year = {2026},
  journal = {Journal of Medical Genetics},
  doi = {10.1136/jmg-2026-111649},
  url = {https://doi.org/10.1136/jmg-2026-111649}
}

RIS

TY  - JOUR
TI  - Safety and efficacy of AAV-based mini- and micro-dystrophin gene therapies in Duchenne muscular dystrophy: a systematic review and meta-analysis of clinical trials
AU  - Haya Nassour
AU  - Samer A Al_Shbailat
AU  - Arwa Sobhi Ibrahim
AU  - Amal Mohammad Allan
AU  - Mostafa Saad Badie Aboudeeb
AU  - Qais M Alnjoom
AU  - Mohammad Jebril Farhan Alrfou
AU  - George Elian Makhoul
AU  - Mahmoud Ghassan Weis
AU  - Rasha K. Ahmad
AU  - Sara M. Abdelstar
PY  - 2026
JO  - Journal of Medical Genetics
DO  - 10.1136/jmg-2026-111649
UR  - https://doi.org/10.1136/jmg-2026-111649
ER  - 

APA

Nassour, H., Al_Shbailat, S. A., Ibrahim, A. S., Allan, A. M., Aboudeeb, M. S. B., Alnjoom, Q. M., Alrfou, M. J. F., Makhoul, G. E., Weis, M. G., Ahmad, R. K., & Abdelstar, S. M. (2026). Safety and efficacy of AAV-based mini- and micro-dystrophin gene therapies in Duchenne muscular dystrophy: a systematic review and meta-analysis of clinical trials. Journal of Medical Genetics. https://doi.org/10.1136/jmg-2026-111649

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