There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved proof-of-principle success with “silence and replace” gene therapy—an approach that uses a viral vector to silence genes with disease-causing mutations and replace them with healthy genes—to prevent and even reverse disease progression.
This article was originally published on MedicalXpress.com
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