Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia

Recently, clinical trials of adeno-associated virus-mediated replacement therapy have suggested long-term therapeutic effects for several genetic diseases of the liver, including hemophilia. However, there remain concerns regarding decreased therapeutic effects when the liver is regenerated or when...

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Detalles Bibliográficos
Autores principales: Lee, Jeong Hyeon, Oh, Hye-Kyung, Choi, Beom Seok, Lee, Ho Hyeon, Lee, Kyu Jun, Kim, Un Gi, Lee, Jina, Lee, Hyerim, Lee, Geon Seong, Ahn, Se Jun, Han, Jeong Pil, Kim, Seokjoong, Yeom, Su Cheong, Song, Dong Woo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9403902/
https://www.ncbi.nlm.nih.gov/pubmed/36090746
http://dx.doi.org/10.1016/j.omtn.2022.08.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9403902/
https://www.ncbi.nlm.nih.gov/pubmed/36090746
http://dx.doi.org/10.1016/j.omtn.2022.08.002

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