Sequencing data from Massachusetts General Hospital shows Cas9 integration into the genome, highlighting a serious hazard in gene-editing therapeutics

Sandeep Chakraborty

doi:https://doi.org/10.12688/f1000research.20744.1

doi.org/10.12688/f1000research.20744.1

Sequencing data from Massachusetts General Hospital shows Cas9 integration into the genome, highlighting a serious hazard in gene-editing therapeutics

Sandeep Chakraborty

14

F1000Research

Volume: 8, Pages: 1846 - 1846

Published: Nov 4, 2019

Abstract

The ability to edit a specific gene within our genomes using guided-nucleases (Cas9/ZFN/TALEN - CaZiTa) presents huge opportunities for curing many genetic disorders. Delivery of this ‘drug’ within cells is a critical step for such therapies. The ability of recombinant adeno-associated virus (rAAV) to enter cells makes it a perfect choice as a vector for gene therapy. A plasmid comprising the rAAV, the CaZiTa, guide RNAs (for CRISPR) is...

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Paper Details

Title

Sequencing data from Massachusetts General Hospital shows Cas9 integration into the genome, highlighting a serious hazard in gene-editing therapeutics

DOI

doi.org/10.12688/f1000research.20744.1

Published Date

Nov 4, 2019

Journal

F1000Research

Volume

8

Pages

1846 - 1846

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