Use of gene-editing tool corrects Pompe mutations in patient cells

Researchers in the U.S. used a gene-editing tool in the lab to correct mutations known to cause infantile-onset Pompe disease, and this enabled patient-derived cells to produce a working version of the acid alpha-glucosidase (GAA) enzyme they lacked. The approach “may offer an efficacious, long-term therapy,” the researchers wrote,…

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…

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…