CRISPR-Cas9 gene editing revolutionized biological and biomedical research virtually overnight. The versatile tool has been used to improve crop yields and cure previously uncurable conditions like sickle cell anemia. In May 2025, the Nobel Prize-winning technology brought about another major biomedical breakthrough, as Kiran Musunuru and Rebecca Ahrens-Nicklas introduced the first personalized CRISPR gene editing... The post The CRISPR revolution continues appeared first on Cold Spring Harbo...| Cold Spring Harbor Laboratory
Neurofibromatosis type 1 (NF1) is a rare genetic condition that affects approximately 100,000 Americans. Children diagnosed with the disorder often have motor delays and learning disabilities. Many are also diagnosed with autism or brain tumors. While significant progress has been made in understanding the biology of NF1 in animal models, translating these findings into clinical... The post NF1: Scientific insight and systemic change appeared first on Cold Spring Harbor Laboratory.| Cold Spring Harbor Laboratory
The discovery of an unknown organelle inside our cells could open the door to new treatments for devastating inherited diseases.| UVA Health Newsroom