Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo

Guide RNAs offer programmability for CRISPR-Cas9 genome editing but also add challenges for delivery. Chemical modification, which has been key to the success of oligonucleotide therapeutics, can enhance the stability, distribution, cellular uptake, and safety of nucleic acids. Previously, we engine...

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Detalles Bibliográficos
Autores principales: Zhang, Han, Kelly, Karen, Lee, Jonathan, Echeverria, Dimas, Cooper, David, Panwala, Rebecca, Chen, Zexiang, Gaston, Nicholas, Newby, Gregory A., Xie, Jun, Liu, David R., Gao, Guangping, Wolfe, Scot A., Khvorova, Anastasia, Watts, Jonathan K., Sontheimer, Erik J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10055305/
https://www.ncbi.nlm.nih.gov/pubmed/36993169
http://dx.doi.org/10.1101/2023.03.20.533459

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