This photo shows green dystrophin protein restored by dual-Cas3 in skeletal muscle derived from DMD patient iPSCs. Credit: Akitsu Hotta Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. On August 24...
Resting state functional magnetic resonance imaging data analysis pipeline. Three cohorts of mice were scanned in the 9.4 Tesla Varian MRI scanner with the bore size of 31 cm. FMRI raw data were acquired using the 2D gradient echo-based echo planer imaging (GE-EPI)...
The first proof of an effective gene therapy for a metabolic disease of the liver is reported in patients with Crigler-Najjar syndrome. No serious adverse events were reported in the phase I/II study evaluating the safety and efficacy of a gene therapy for patients...
Researchers from New York University, Columbia University, and the New York Genome Center have developed TIGER (Targeted Inhibition of Gene Expression via guide RNA design), a deep learning model that combines CRISPR technology with artificial intelligence to...
Unveiling a groundbreaking discovery, a team of researchers led by the Feng Zhang at the Broad Institute has discovered a eukaryotic programmable RNA-guided system. Published in the journal Nature, their study unveils a system centered around the Fanzor protein. The...