Therapeutic Gene-Editing in Alzheimer’s disease
Principal Investigator
Subhojit Roy
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.
Grants
Related Grants
Alzheimer's Disease Research
Regulatory Mechanisms Underlying Endosomal Targeting of SORL1
Active Dates
January 01, 2025 - December 31, 2026
Principal Investigator
Olav Andersen, PhD
Regulatory Mechanisms Underlying Endosomal Targeting of SORL1
Active Dates
January 01, 2025 - December 31, 2026

Principal Investigator
Olav Andersen, PhD
Alzheimer's Disease Research
The Role of DYRK1A in Altered Microglia Biology in a Cellular Model of Alzheimer’s Disease in Down Syndrome
Active Dates
January 01, 2025 - December 31, 2027
Principal Investigator
Frances Wiseman, PhD
The Role of DYRK1A in Altered Microglia Biology in a Cellular Model of Alzheimer’s Disease in Down Syndrome
Active Dates
January 01, 2025 - December 31, 2027

Principal Investigator
Frances Wiseman, PhD
Alzheimer's Disease Research
Synergistic Effects of Biological Sex and Sleep Loss in an AD Mouse Model
Active Dates
January 01, 2025 - December 31, 2026
Principal Investigator
Mallar Chakravarty, PhD
Synergistic Effects of Biological Sex and Sleep Loss in an AD Mouse Model
Active Dates
January 01, 2025 - December 31, 2026
Principal Investigator
Mallar Chakravarty, PhD