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An American research team led by Jennifer Doudna from Innovative Genomics Institute at University of California, Berkeley, has demonstrated genome editing in the mouse brain through transient delivery of minimally immunogenic CRISPR-Cas9 ribonucleoproteins (RNPs). The research aimed to overcome viral vector-based delivery - including cargo capacity, immunogenicity, and cost - by testing the ability of cell penetrant Cas9 RNPs to edit the mouse striatum with a convection-enhanced delivery system. Results demonstrated that the transient Cas9 RNPs showed comparable editing of neurons and reduced adaptive immune responses relative to one formulation of Cas9 delivered using AAV serotype 9. #crispr #geneediting #geneomeediting #crisprmedicine #brain #centralnervoussystem  https://lnkd.in/evcgFXaq

Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs

Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs

sciencedirect.com

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