Gene therapy shows promise for treating Niemann-Pick disease type C1 | National Institutes of Health (NIH)

For the first time, National Institutes of Health researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1). The gene therapy involved inserting a functional copy of the NPC1 gene into mice with the disease; the treated animals were … Continue reading Gene therapy shows promise for treating Niemann-Pick disease type C1 | National Institutes of Health (NIH)

Source: Biosingularity - November 13, 2016 Category: Research Authors: Derya Tags: Biotechnology Source Type: blogs