MPS IIIA: GENE THERAPY FOR BRAIN AND BODY (SANFILIPPO)
SciBX By Michael J. Haas, Senior Writer
Less than a year after reporting preclinical proof of concept for a systemically
delivered mucopolysaccharidosis IIIA gene therapy, researchers
at the Autonomous University of Barcelona have shown that intracerebral
delivery can substantially improve efficacy in the CNS and treat
symptoms in peripheral organs.1 ESTEVE., who partially funded the
work, is now raising funds to test the approach in the clinic.
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