Gene therapy is a powerful developing technology that has the potential to address myriad diseases. For example, Huntington’s disease, a neurodegenerative disorder, is caused by a mutation in a single gene, and if researchers could go into specific cells and correct that defect, theoretically those cells could regain normal function. […]
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A novel computational system created by scientists from the University of Pittsburgh University of Medication identifies best-performing viral vectors that could deliver gene therapies to the retina with most performance and precision. The technologies, described in a paper revealed now in the journal eLife, streamlines advancement of gene remedy strategies […]