CRISPR

Computer Simulations Could Help Design Gene Therapies for Pompe, Study Suggests

A CRISPR gene-editing approach was used to correct two distinct Pompe disease mutations and to develop computer simulations for optimizing gene therapies. These computer models may help scientists develop and test gene-editing strategies for diseases, such as Pompe, that are polygenic, or caused by more…

Gene Therapy Takes Center Stage at 2019 NORD Summit

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…