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Families of SMA has Funded the Following Projects on Gene Therapy Research: 1) In 2003, to Dr. Terence Flotte at the University of Florida for $242,602 for "Recombiant AAV Gene Therpy for Spinal Muscular Atrophy". 2) In 2006, to Dr. Christian Lorson at University of Missouri for $124,072 for "Development of SMN rAAV Vectors". 3) In 2010, to Dr. Brian Kaspar at Nationwide Children's Hospital at Ohio State University for $100,000 for "Optimizing Titer and Window of Opportunity for Targeting Motor Neurons via an AAV9 Vector in Newborn Non-human Primates". 4) In 2012, to Dr. Brian Kaspar at Nationwide Children's Hospital at Ohio State University for up to $750,000 after meeting pre-determined project milestones for “IND Enabling Studies for a CNS Delivered Gene Therapy for Spinal Muscular Atrophy". Total Possible Funding for the Gene Therapy of $1,216,674.
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