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Global Translational Medicine AAV vectors in gene therapy: Advances and applications

and transfer of therapeutic genes in gene therapies for 7. Asaad W, Volos P, Maksimov D, et al. AAV genome
cancer, neurodegenerative diseases, retinal diseases, and modification for efficient AAV production. Heliyon.
COVID-19. 13-16 In light of our growing understanding 2023;9(4):e15071.
of viral biology and the availability of diverse platforms, doi: 10.1016/j.heliyon.2023.e15071
it is essential to optimize AAV vector delivery system in 8. Tan F, Dong Y, Qi J, Yu W, Chai R. Artificial intelligence-
terms of capsid properties, production yield, packaging based approaches for AAV vector engineering. Adv Sci
efficiency, immune response, biodistribution potential, (Weinh). 2025;12(9):e2411062.
and transduction safety, which are critical for the successful
application and development of clinically approved doi: 10.1002/advs.202411062
therapy. Consequently, future research should focus on 9. Maguire AM, Bennett J, Aleman EM, Leroy BP, Aleman TS.
creating AI-driven tools for AAV capsid engineering and Clinical perspective: Treating rpe65-associated retinal
packaging capacity/immune response prediction methods dystrophy. Mol Ther. 2021;29(2):442-463.
evolving from the current basic programming to support doi: 10.1016/j.ymthe.2020.11.029
the learning of more advanced concepts.
10. Alsalloum A, Gornostal E, Mingaleva N, et al. A comparative
Conflict of interest analysis of models for AAV-mediated gene therapy for
inherited retinal diseases. Cells. 2024;13(20):1706.
Ling Yin is the Guest Editor of this special issue. The author doi: 10.3390/cells13201706
declared that he has no known competing financial interests
or personal relationships that could have influenced the 11. Pierce EA, Bennett J. The status of RPE65 gene therapy
work reported in this paper. trials: Safety and efficacy. Cold Spring Harb Perspect Med.
2015;5:a017285.
References doi: 10.1101/cshperspect.a017285
1. Tang R, Xu Z. Gene therapy: A double-edged sword with 12. Ogbonmide T, Rathore R, Rangrej SB, et al. Gene therapy
great powers. Mol Cell Biochem. 2020;474(1-2):73-81. for spinal muscular atrophy (SMA): A review of current
challenges and safety considerations for onasemnogene
doi: 10.1007/s11010-020-03834-3
abeparvovec (Zolgensma). Cureus. 2023;15(3):e36197.
2. Rind DM. The FDA and gene therapy for duchenne muscular doi: 10.7759/cureus.36197
dystrophy. JAMA. 2024;331(20):1705-1706.
13. Yang H, Qing K, Keeler GD, et al. Enhanced transduction
doi: 10.1001/jama.2024.5613
of human hematopoietic stem cells by AAV6 vectors:
3. Singh A, Irfan H, Fatima E, Nazir Z, Verma A, Akilimali A. Implications in gene therapy and genome editing. Mol Ther
Revolutionary breakthrough: FDA approves CASGEVY, the Nucleic Acids. 2020;20:451-458.
first CRISPR/cas9 gene therapy for sickle cell disease. Ann doi: 10.1016/j.omtn.2020.03.009
Med Surg (Lond). 2024;86(8):4555-4559.
14. Yin L, Keeler GD, Zhang Y, et al. AAV3-mirna vectors for
doi: 10.1097/MS9.0000000000002146
growth suppression of human hepatocellular carcinoma
4. Anguela XM, High KA. Hemophilia b and gene therapy: cells in vitro and human liver tumors in a murine xenograft
A new chapter with etranacogene dezaparvovec. Blood Adv. model in vivo. Gene Ther. 2021;28:422-434.
2024;8(7):1796-1803.
doi: 10.1038/s41434-020-0140-1
doi: 10.1182/bloodadvances.2023010511
15. Zhu P, Dyka F, Ma X, et al. Disease mechanisms of x-linked
5. Gardin A, Ronzitti G. Current limitations of gene cone dystrophy caused by missense mutations in the red and
therapy for rare pediatric diseases: Lessons learned from green cone opsins. FASEB J. 2021;35(10):e21927.
clinical experience with AAV vectors. Arch Pediatr. doi: 10.1096/fj.202101066R
2023;30(8S1):8S46-8S52.
16. Glazkova DV, Bogoslovskaya EV, Urusov FA, et al.
doi: 10.1016/S0929-693X(23)00227-0
Generation of sars-cov-2 mouse model by transient
6. Naso MF, Tomkowicz B, Perry WL 3 , Strohl WR. Adeno- expression of the human ace2 gene mediated by
rd
associated virus (AAV) as a vector for gene therapy. intranasal administration of aav-hace2. Mol Biol (Mosk).
BioDrugs. 2017;31(4):317-334. 2022;56:705-712.
doi: 10.1007/s40259-017-0234-5 doi: 10.1134/S0026893322050065

Volume 4 Issue 2 (2025) 2 doi: 10.36922/GTM025120026