Coming of age: an overview of the growing toolbox for gene editing and its use in CGT applications

Cell & Gene Therapy Insights 2024; 10(9), 1221–1236

DOI: 10.18609/cgti.2024.139

Published: 7 October
Review
Christina Fuentes, Jacob Staudhammer, Madison Pope, Scott Cross

Genome editing has the potential to expand the number and types of indications that can be targeted by cell and gene therapies. In recent years, there have been significant advancements in the genome editing tools, technologies, delivery methods, and analytical techniques available to developers. This review covers available genome editing tools, their utility, safety profile, as well as high-level manufacturing and regulatory strategy considerations when using these advanced technologies.