When Michael Pirovolakis received personalized gene therapy in a single-patient clinical trial at SickKids Hospital in March 2022, the course of his disease changed dramatically. Michael...
Researchers at Vanderbilt University Medical Center (VUMC) are touting data from a multicenter, international Phase 2 clinical trial demonstrating a new treatment for sickle cell disease...
Researchers at the Icahn School of Medicine at Mount Sinai have identified a neurodevelopmental disorder caused by a single gene mutation that affects tens of thousands...
In a step forward in the development of genetic medicines, researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University...
Obesity and related metabolic disorders such as type 2 diabetes are major public health concerns. Nearly one in four people are obese, according to the Organization...
CRISPR-Cas9 is widely used to edit genomes by studying genes of interest and altering disease-associated genes. However, this process is accompanied by side effects such as...
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