Gene editing possible for inherited retinal diseases

By The Science Advisory Board staff writers

September 26, 2022 -- Precision genome editing agents can enable gene correction and disease rescue in inherited retinal diseases (IRDs), according to University of California, Irvine researchers.

These disorders affect approximately 1 in 3,000 individuals worldwide and reduce quality of life. IRDs are caused by gene mutations that are critical for the development and/or function of the retina or RPE, and more than 270 causative genes have been identified.

The researchers argue in their article that precision genome editing agents, including base editors and prime editors, have enabled precise gene correction and disease rescue in multiple preclinical models of genetic disorders (Proceedings of the National Academy of Sciences, September 19, 2022). Why not use them for IRDs?

The paper stresses there is hope that in vivo gene editing will be the future treatment paradigm for IRDs. The authors said there will be an increasing number of clinical trials for targeting IRDs and any mutation that causes them will be amenable to treatment with precision gene editing.

"Precision medicine for IRDs has a promising outlook, as basic science has consistently led to the development of therapeutic tools to target patient-specific genetic mutations," the authors wrote. "The results of initial clinical trials, that use in vivo gene editing to treat IRDs, will be essential for informing the design and translation of future precision genome editing therapies."

Mammoth Biosciences looks to build next-generation CRISPR toolbox
This month marks the 10th anniversary of the development of CRISPR as a genome-editing tool. While the technology is fairly new, it has made major strides...
Berkeley Lab offers CRAGE kit for genome engineering
Lawrence Berkeley National Laboratory (Berkeley Lab) is offering a trial kit for its chassis-independent, recombinase-assisted, genome engineering (CRAGE)...
11 new developments in cell and gene therapy
There have been a number of recent developments in the cell and gene therapy field. Bruce Carlson, publisher of Cell and Gene Therapy Business Outlook,...
Investment into regenerative medicine grows alongside company collaborations
Regenerative medicine is a relatively new area of medicine that involves significant research into how to harness the body's own healing mechanisms as...
Gene therapy research collaborations gain traction for next-gen medicines
Research collaborations are becoming increasingly more common, allowing companies to pool existing resources to advance innovations in the field of medicine....

Copyright © 2022 scienceboard.net


Conferences
Connect
Science Advisory Board on LinkedIn
Science Advisory Board on Facebook
Science Advisory Board on Twitter