The potential of precise genome editing in the treatment of hereditary retinal diseases

In a new paper, researchers at the University of California, Irvine, explain how precision genome editing agents enable precise gene correction and disease rescue
in hereditary retinal diseases (IRDs).

This paper describes the current preclinical success and clinical genome editing methods for the treatment of hereditary retinal degenerative diseases, and emphasizes that in vivo gene editing is expected to become the treatment paradigm
for future retinal degenerative diseases.

Dr.

This paper describes the progress of using genome editing to treat IRDs and important considerations
for robust clinical translation.

"More and more CRISPR-based treatments are being tested in clinical trials," Palczewski said
.

Programmable CRISPR-Cas nuclease is an effective tool for gene destruction, but in most therapeutic settings, they are not suitable for precisely correcting pathogenic mutations
.

Hereditary retinal diseases (IRDs) are a group of inherited heterogeneous blinding diseases characterized by progressive degradation
of photoreceptors and retinitis pigment epithelium (RPE).

Over the past 20 years, significant advances in gene therapy have brought new hope
for the successful treatment of these IRDs.

Palczewski said: "Precision medicine for IRDs has a good prospect because basic science has been leading the development of therapeutic tools aimed at targeting patient-specific genetic mutations
.

Susie Suh, Elliot H.