Targeted Knockout of the Vegfa Gene in the Retina by Subretinal Injection of RNP Complexes Containing Cas9 Protein and Modified sgRNAs

The therapeutic effect of retinal gene therapy using CRISPR/Cas9-mediated genome editing and knockout applications is dependent on efficient and safe delivery of gene-modifying tool kits. Recently, transient administration of single guide RNAs (sgRNAs) and SpCas9 proteins delivered as ribonucleoprot...

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Bibliographic Details
Published in:Mol Ther
Main Authors: Holmgaard, Andreas Braae, Askou, Anne Louise, Jensen, Emilie Grarup, Alsing, Sidsel, Bak, Rasmus O., Mikkelsen, Jacob Giehm, Corydon, Thomas J.
Format: Artigo
Language:Inglês
Published: American Society of Gene & Cell Therapy 2021
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC7791085/
https://ncbi.nlm.nih.gov/pubmed/33022212
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.09.032
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