In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy

Hemophilia is a hereditary disease that remains incurable. Although innovative treatments such as gene therapy or bispecific antibody therapy have been introduced, substantial unmet needs still exist with respect to achieving long-lasting therapeutic effects and treatment options for inhibitor patie...

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Detalles Bibliográficos
Autores principales: Han, Jeong Pil, Kim, MinJeong, Choi, Beom Seok, Lee, Jeong Hyeon, Lee, Geon Seong, Jeong, Michaela, Lee, Yeji, Kim, Eun-Ah, Oh, Hye-Kyung, Go, Nanyeong, Lee, Hyerim, Lee, Kyu Jun, Kim, Un Gi, Lee, Jae Young, Kim, Seokjoong, Chang, Jun, Lee, Hyukjin, Song, Dong Woo, Yeom, Su Cheong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2022
Materias:
Biomedicine and Life Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8782450/
https://www.ncbi.nlm.nih.gov/pubmed/35061543
http://dx.doi.org/10.1126/sciadv.abj6901

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