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Gene Therapy Lab

Gene therapy can modify mutations in genes or deliver an extra, healthy copy of an allele to treat inherited forms of blindness. Dr. Mahajan was a surgeon in the recent trial for gene therapy using a viral vector that successfully rescued vision loss in patients with Leber Congenital Amaurosis. The development of pre-clinical models to test gene therapy approaches for other forms of blindness, as well as testing gene therapy treatments in these pre-clinical animal models, is necessary and important to lead to clinical trials for human patients.

We are treating human patient stem cells and translational animal models with gene therapy approaches. We are also developing animal models for various inherited retinal degenerative diseases by introducing human genes or human mutations into the animals.

Pluripotent stem cells are made in the lab from patient volunteers with retinal degenerations. We then use gene editing (CRISPR) to correct the patient mutations in their own cells. Our lab was the first to correct a human blindness gene in human stem cells. These gene-corrected cells can then be used to test therapies and create transplants that replace diseased retinas.

Projects

Fixing faulty genes.
Direct modification of DNA in living cells.
Gene therapy correction in pre-clinical models of retinal disease.

News

Aug 11 2020 | Posted In: 20/20 Blog
Palo Alto, CA — Gene therapy has become a viable option for people with inherited eye disease, but because it is still a novel treatment, patient progress following gene therapy often relies on subjective measurements
Apr 20 2020 | Posted In: 20/20 Blog
Palo Alto, CA — Is there something to feed retinal cells that can give them the energy to withstand gene mutations that make them sick?
Oct 16 2019 | Posted In: 20/20 Blog
Palo Alto, CA — A gene therapy clinical trial for Dry Age Related Macular Degeneration (AMD) is beginning at the Byers Eye Institute under the direction of Vinit Mahajan M.D., Ph.D., associate professor and Vice Chair for Research in Ophthalm
Oct 14 2019 | Posted In: 20/20 Blog
Palo Alto, CA – Dry macular degeneration is a common eye condition that usually occurs in people over 50. It is characterized by blurring of the central vision along with a decreased ability to see colors and fine details.
Sep 16 2019 | Posted In: 20/20 Blog
Palo Alto, CA – The Molecular Surgery Program in Stanford’s Ophthalmology Department held the first Bay Area Genetic Retinal Disease Case Conference at the 

Publications

CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa: A Brief Methodology, Wu, Wen-Hsuan, Tsai Yi-Ting, Justus Sally, Cho Galaxy Y., Sengillo Jesse D., Xu Yu, Cabral Thiago, Lin Chyuan-Sheng, Bassuk Alexander G., Mahajan Vinit B., et al. , Retinal Gene Therapy, p.191–205, (2018)
Translation of CRISPR Genome Surgery to the Bedside for Retinal Diseases., Xu, Christine L., Cho Galaxy Y., Sengillo Jesse D., Park Karen S., Mahajan Vinit B., and Tsang Stephen H. , Front Cell Dev Biol, 2018, Volume 6, p.46, (2018)
Autologous stem cell therapy for inherited and acquired retinal disease, Apatoff, Mary Ben L., Sengillo Jesse D., White Eugenia C., Bakhoum Mathieu F., Bassuk Alexander G., Mahajan Vinit B., and Tsang Stephen H. , Regenerative medicine, (2018)
CRISPR-Mediated Ophthalmic Genome Surgery, Cho, Galaxy Y., Abdulla Yazeed, Sengillo Jesse D., Justus Sally, Schaefer Kellie A., Bassuk Alexander G., Tsang Stephen H., and Mahajan Vinit B. , Current ophthalmology reports, Volume 5, p.199–206, (2017)
CRISPR-Cas Genome Surgery in Ophthalmology, DiCarlo, James E., Sengillo Jesse D., Justus Sally, Cabral Thiago, Tsang Stephen H., and Mahajan Vinit B. , Translational vision science & technology, Volume 6, p.13–13, (2017)
Retrospective Analysis of Structural Disease Progression in Retinitis Pigmentosa Utilizing Multimodal Imaging, Cabral, Thiago, Sengillo Jesse D., Duong Jimmy K., Justus Sally, Boudreault Katherine, Schuerch Kaspar, Belfort Rubens, Mahajan Vinit B., Sparrow Janet R., and Tsang Stephen H. , Scientific reports, Volume 7, Number 1, p.10347, (2017)