In a recently published study, Nature Biomedical EngineeringA group of researchers optimized prime editing (PE) to efficiently correct the cystic fibrosis (CF) transmembrane conductance regulator (CFTR)...
Improving the accuracy of gene editing “We have identified what could be the next wave of hereditary anemia treatments,” said co-author Mitchell Weiss, MD, Ph.D., St....
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...
In a ‘bench-to-bedside’ collaboration between UCL and Great Ormond Street Hospital for Children, base-edited T cells were administered to patients with relapsed T-cell leukemia in the...
Ann & Robert H. Lully Children’s Hospital of Chicago’s Stanley Man Children’s Research Institute, Youyang Zhao, PhD’s lab, is working to deliver genome-editing technology, including CRISPR...
In one type of rare hereditary disorder that affects the control of body movements, scientists have discovered mutations in enzymes that impair communication between neurons. Walk...
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