Maanas Memorial Fund Supports 2015 Research Grant

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Last Updated: Tuesday, 23 February 2016 09:20

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
Institution:  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

Lemonade & Football for Hope

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Last Updated: Tuesday, 01 September 2015 10:34

Yard Sale to Benefit Hope

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Last Updated: Tuesday, 01 September 2015 13:50

Saturday, September 26th and
Sunday, September 27th

12 Quail Lane

Limington, Maine 04049

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Aurora has taught us about love.
Not the gentle, butterfly love of spring,
but the excruciating, tenacious love
of deep winter.
She has taught us how to hold tightly together
through this frigid madness. 
To weld our hands and hearts 
into something stronger than before.
An iron bond of desperate, tragic, love.
This is her gift.
To forge her family
in the heat of her heart,
handing us a mirror,
to show us our own strength.

LipSync4ACure

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Last Updated: Tuesday, 25 August 2015 10:13

A new challenge that is easy (and drier than the other well-known challenge) has been issued by the families of NTSAD!

The rules:

1. Lip sync to your favorite song for one minute;

2. Challenge three people to do the same and/or make a gift to NTSAD here.

3. Share on the #LipSync4ACure page and your Facebook page.

4. Feel good knowing you've spread hope and had a little fun at the same time!

Extra Challenge: 

We want to get Jimmy Fallon's attention and challenge him to #LipSync4ACure on NBC's "Tonight Show!"