ELK GROVE VILLAGE, Ill., Nov. 5, 2014 /PRNewswire-USNewswire/ -- A manuscript from the laboratory of Dr. Brian Kaspar of Nationwide Childrens Hospital was recently published in the journal Molecular Therapy. The paper, Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA-a dose response study in mice and nonhuman primates is the first publication resulting from a groundbreaking collaboration between Cure SMA, the National Institute of Neurological Disorders and Stroke (NINDS), and Dr. Kaspar. Beginning in 2010, Cure SMA made a series of grants to Dr. Kaspar to study gene therapy, also called gene transfer. Spinal muscular atrophy (SMA) is caused by a mutation in the survival motor neuron 1 gene (SMN1). Because of this mutation, the individual does not produce enough survival motor neuron (SMN) protein. Gene transfer may increase SMN levels by using a virus, called a vector, to deliver the SMN1 gene to affected cells. Kaspars laboratory discovered that Adeno-associated virus serotype 9 (AAV9) had the unique ability to cross the blood brain barrier and the Blood-Cerebrospinal Fluid Barrier (CSF). Dr. Kaspar and his team have studied two approaches for SMA: an injection into a vein, a process known as systemic delivery which is currently in … Continue reading
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