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Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Maanas Memorial Fund Supports 2015 Research Grant

NTSAD Research Initiative Grant Supported
July 2015

About the Maanas Memorial Fund

Ria Datla, 17, in memory of her younger brother who died due to Sandhoff disease, performed  the Kuchipudi "Rangapravesam" -- a classical Indian dance from Andhra Pradesh.   Her goal was to raise  $10,000 for Sandhoff research.

“The Datla family’s support is crucial to our effort to develop a less invasive gene therapy treatment through the bloodstream. In addition to the financial gift, we’re so grateful for their moral support and trust. We share Ria’s goal ‘to remove fear and replace it with confidence,’ and we’ll do our best to honor Maanas in this work.” 

…Douglas R. Martin, PhD.

NTSAD Research Initiative

Project:  Intravascular Gene Therapy for Feline GM2 Gangiosidois
Principal Investigator: 
Douglas R. Martin, PhD
Auburn University

Goals of the proposal:

  • Optimize intravascular (IV) gene therapy to treat both central nervous system (CNS) and peripheral (rest of body) manifestations of feline Sandhoff disease (SD).
  • Conduct short-term (6 week) studies to compare different delivery routes (i.e. carotid artery and cephalic vein) for optimal therapeutic effect in the CNS and minimized vector levels in the periphery.
  • After selecting optimal delivery route, the therapeutic effect of pre-treating cats with mannitol to temporarily open the blood brain barrier and possibly permit use of a lower dose of vector for long-term therapy will be evaluated.

Impact of research:

NTSAD has previously supported efforts of the Tay-Sachs Gene Therapy Consortium to develop an effective gene therapy protocol for TSD and SD. Previous studies with direct intracranial injection of Adeno-associated virus (AAV) vectors expressing hexosaminidase dramatically increased the life span and quality of life in SD cats. While these proof-of-concept studies continue to support human application of intracranial gene therapy for TSD and SD, it is desirable to develop a less invasive and presumably safer approach that will also better address peripheral disease.

Read more: Maanas Memorial Fund

Maanas Memorial Fund