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ISABEL JURADO FOUNDATION

ISABEL JURADO FOUNDATION
CROWDRISE : Jun 08, 2014
Tax ID: 20-3903321
BASED: Rock Hill, SC, United States

CHARITY WEBSITE: http://www.isabelfoundation.org

ISABEL JURADO FOUNDATION

Sanfilippo Gene Therapy

Take a "Stand" Against Sanfilippo Syndrome

Help for Today, Hope for Tomorrow. The Isabel Jurado Foundation’s mission is to fund research for Sanfilippo Syndrome and provide family assistance for all MPS diseases. It was founded in honor and memory of those children, past and present, who have suffered the deadly impact of Sanfilippo Syndrome. 

All funds raised from our virtual Purple Lemonade Stand will be used to fund clinical trials for Sanfilippo Type A and B gene therapy at Nationwide Children's Hospital in OH. 

About the Research 

The Research Institute at Nationwide Children’s Hospital - AAV9 Gene Therapy in Patients with Sanfilippo Type A and B

Investigators: Dr. Kevin Flanigan, Dr. Haiyan Fu, Dr. Douglas McCarty

Research at NCH is poised to begin a phase I/II gene therapy clinical trial for children with types A and B as early as December 2014.  An AAV9 vector containing a functioning copy of the gene is injected into the patient intravenously. In animal models, the therapy resulted inwidespread expression of the gene, a clearance of the stored material, improved cognitive function and increased lifespan. An investment of approximately $4 million dollars is needed to bring this research to trial.

Tax ID: 20-3903321 • http://www.isabelfoundation.org

Fundraisers

Purple Lemonade for Isabel Jurado Foundation

Purple Lemonade for Isabel J…

Amount Raised:

$788

 

3% Raised of $25,000 Goal