Therapeutic Gene-Editing in Alzheimer’s disease
Principal Investigator
Subhojit Roy, MD, PhD
The Regents of the University of California, San Diego
La Jolla, CA, United States
About the Research Project
Program
Award Type
Standard
Award Amount
$300,000
Active Dates
July 01, 2023 - June 30, 2026
Grant ID
A2023023S
Goals
Our overall goal is to take a CRISPR based gene therapy for Alzheimer’s disease to the clinic.
Summary
Our overall goal is to take a CRISPR based gene therapy for Alzheimer’s disease to the clinic. Broadly speaking, our approach restores the physiologic balance of the amyloid pathway – decreasing neurotoxic protein fragments, while promoting neuroprotective protein fragments. There are over a 100 ongoing clinical trials worldwide using CRISPRs, and the results so far with non-neurologic disorders have shown unprecedented (almost 100%) efficacy. Our broad vision is to apply CRISPRs for devastating neurologic illnesses like Alzheimer’s disease.
Unique and Innovative
If successful, this will be the first CRISPR based therapeutic for any neurologic disease.
Foreseeable Benefits
Upon completion, we will be closer to a novel “one and done” CRISPR-based gene therapy for Alzheimer’s disease.
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