Event-Triggered Gene Therapy for ALS: Smart Release of Therapeutics in the Brain
Abstract
Our proposal will focus on special enzymes, called proteases that digest proteins and change the cellular environment surrounding the motor neurons in the spinal cord and the brain. Many of pathological events in the spinal cord and the brain can be traced to the local proinflammatory processes that trigger several downstream mechanisms including the activation of proteases, enzymes that cleave proteins in and around the cells of the brain. We propose a strategy to develop transmembrane proteins that can be delivered to the spinal cord and the brain, specifically designed to release therapeutic proteins at the sites affected by the disease, after being cleaved by proteases that are activated during ALS. Our team has developed a way to change these proteases so that instead of being harmful they can now release therapeutic proteins. Once inserted into the cell membrane of spinal cord and brain cells, these sensors can be activated by the injury, to release therapeutics in the spinal cord and the brain to avert loss of motor neurons and prevent the progression of ALS.
Document Details
- Document Type
- Technical Report
- Publication Date
- Mar 01, 2024
- Accession Number
- AD1227972
Entities
People
- Ljubomir Kojic
- Max Cynader
Organizations
- University of British Columbia