Gene therapy

Adult stem cell-derived organoids closely resemble their tissue of origin. This Review discusses recent developments in CRISPR-mediated genome engineering and its application using adult-stem-cell-derived organoids in the construction of isogenic disease models and for clinical gene repair.

In this Review, the authors discuss gene therapy involving the use of recombinant adeno-associated virus vectors for the treatment of inherited liver diseases, including ongoing clinical trials that are producing promising results.

The design of prime editing guide RNAs is optimized by deep learning.

CRISPR-Cas tools have shown exceptional promise in genome engineering over the past decade. Here the authors review the development of CRISPR-Cas9/Cas12/Cas13 nucleases, DNA base editors, prime editors, and RNA base editors, as well as their editing precision, off-target effects, and clinical considerations.