Award
National Institute of Neurological Disorders and Stroke (NINDS) and National Eye Institute (NEI) 1R44NS141514-01A1
Treatment of GM1 gangliosidosis CNS manifestations by AAV systemic expression of lectin-enhanced enzyme
Recipient
STATE UNIVERSITY
Award Amount
$1,500,000.00
Ceiling
$1,500,000.00
Awarded
September 15, 2025
Identifier
1R44NS141514-01A1
NIH awarded a grant for research on gene therapy targeting GM1 gangliosidosis CNS manifestations using AAV systemic expression of lectin-enhanced enzyme, with a funding amount of $1,500,000 for the period 2025-09-15 to 2027-08-30.
Description
This is a NIH grant opportunity for research on gene therapy for GM1 gangliosidosis. The project aims to develop systemic treatment options crossing the blood-brain barrier, utilizing AAV-based gene therapy and lectin-enhanced enzymes. The project involves multiple NIH institutes and has a multi-year funding plan.