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Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning

Therapeutic payload delivery to the central nervous system (CNS) remains a major challenge in gene therapy. Recent studies using function-driven evolution of adeno-associated virus (AAV) vectors have successfully identified engineered capsids with improved blood-brain barrier (BBB) penetration and C...

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Detalles Bibliográficos
Autores principales: Nonnenmacher, Mathieu, Wang, Wei, Child, Matthew A., Ren, Xiao-Qin, Huang, Carol, Ren, Amy Zhen, Tocci, Jenna, Chen, Qingmin, Bittner, Kelsey, Tyson, Katherine, Pande, Nilesh, Chung, Charlotte Hiu-Yan, Paul, Steven M., Hou, Jay
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7841218/
https://www.ncbi.nlm.nih.gov/pubmed/33553485
http://dx.doi.org/10.1016/j.omtm.2020.12.006