Rare genetic diseases are challenging for patients and their families—made all the more overwhelming because symptoms tend to appear soon after birth. To date, there haven’t been many reliable treatment options for these babies. The few that do exist involve invasive and risky procedures that don’t often have a high rate of success. But there … Read more
Kidney disease can lead to high blood pressure and infections Mohammed Haneffaa Nizamudeen/Getty Images The damage to the body caused by the most common type of inherited kidney disease was thought to be irreversible. But now, animal studies suggest that correcting the responsible mutations via Crispr gene editing can reverse at least some of it. … Read more
Baby KJ after a gene-editing infusion with researchers Rebecca Ahrens-Nicklas and Kiran Musunuru Children’s Hospital of Philadelphia A baby boy with a life-threatening genetic condition has become the first person to receive a bespoke Crispr gene-editing treatment, giving a glimpse into what the future of medicine might look like. It’s the first time anyone has … Read more
Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has been challenging. In a study published May 15 in the New England Journal of Medicine and presented at the American Society of Gene and Cell Therapyresearchers led by teams at Children’s Hospital of Philadelphia and University of … Read more
A light micrograph cross-section showing cholesterol-containing plaque in a heart artery NIGEL DOWNER/SCIENCE PHOTO LIBRARY The prospects for a one-time treatment that reduces the risk of heart disease for the rest of a person’s life just got brighter. In an initial trial, a single dose of a CRISPR gene-editing therapy lowered cholesterol levels without any … Read more
The CRISPR protein (blue and pink) is hooked up to an RNA sequence (orange) that guides where to cut DNA (purple) Science Photo Library / Alamy Stock Photo The risk of developing many common conditions could be drastically reduced by making dozens of edits to people’s genomes at the embryo stage, claims a team of … Read more
