In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice

Gene therapy and rebalancing therapy have emerged as promising approaches for treating hemophilia A, but there are limitations, such as temporary efficacy due to individual differences. Genome editing for hemophilia has shown long-term therapeutic potential in preclinical trials. However, a cautious...

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Detalles Bibliográficos
Autores principales: Han, Jeong Pil, Lee, Yeji, Lee, Jeong Hyeon, Chung, Hye Yoon, Lee, Geon Seong, Nam, Yu Ri, Choi, Myeongjin, Moon, Kyoung-Sik, Lee, Haeshin, Lee, Hyukjin, Yeom, Su Cheong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10616378/
https://www.ncbi.nlm.nih.gov/pubmed/37916225
http://dx.doi.org/10.1016/j.omtn.2023.102050

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10616378/
https://www.ncbi.nlm.nih.gov/pubmed/37916225
http://dx.doi.org/10.1016/j.omtn.2023.102050

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