Award
National Institute of Neurological Disorders and Stroke 1R21NS149629-01
Testing a new therapy for SPG4 Hereditary Spastic Paraplegia
Recipient
SCHOOLS OF MEDICINE
Award Amount
$228,688.00
Ceiling
$228,688.00
Awarded
June 10, 2026
Identifier
1R21NS149629-01
NIH-funded research project to develop and validate a new therapy for SPG4 Hereditary Spastic Paraplegia using intrabody vectors and patient-derived motor cortical organoids.
Description
The project involves developing a therapeutic approach using intrabody vectors targeting mutant M1 spastin protein in SPG4-HSP. It utilizes patient-derived hiPSC lines differentiated into motor cortical organoids. The project aims to validate and therapeutically correct the pathogenic accumulation of mutant M1.