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New Targeted Approach Could Prevent Toxicities Associated with Neurological Gene Therapies

November 12, 2020

Via: BioSpace

Researchers from Penn Medicine have developed a new targeted approach that modifies viral vectors and inhibits toxicities in the sensory neurons of dorsal root ganglia (DRG) that commonly occur following the use of gene therapy for neurological diseases.

This strategy will likely have several important research and clinical implications, as investigators in the field have worked tirelessly for years to develop safer and more effective gene therapies for neurological disorders. “We believe that this new approach could improve safety in gene therapy universally,” said lead author Juliette Hordeaux, DVM, Ph.D., senior director of Translational Research in Penn’s Gene Therapy Program, in a statement.

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