A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

Author:

Finn Jonathan D.,Smith Amy Rhoden,Patel Mihir C.,Shaw Lucinda,Youniss Madeleine R.,van Heteren Jane,Dirstine Tanner,Ciullo Corey,Lescarbeau Reynald,Seitzer Jessica,Shah Ruchi R.,Shah Aalok,Ling Dandan,Growe Jacqueline,Pink Melissa,Rohde Ellen,Wood Kristy M.,Salomon William E.,Harrington William F.,Dombrowski Christian,Strapps Walter R.,Chang Yong,Morrissey David V.

Publisher

Elsevier BV

Subject

General Biochemistry, Genetics and Molecular Biology

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4. Quantitative analysis shows that repair of Cas9-induced double-strand DNA breaks is slow and error-prone;Brinkman;bioRxiv,2017

5. ClinicalTrials.gov (2017). APOLLO: the study of an investigational drug, Patisiran (ALN-TTR02), for the treatment of transthyretin (TTR)-mediated amyloidosis. https://clinicaltrials.gov/ct2/show/NCT01960348.

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