By creating gene editors not found in nature, or optimizing existing editors, AI can improve the accuracy, effectiveness, and accessibility of gene editing.

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

This study provides a valuable new resource to investigate the molecular basis of the particular features characterizing the pipefish embryo. The authors found both unique and shared gene expression ...

PRINCETON, N.J., Feb. 03, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that two oral presentations and 20 posters across its development programs will be included at ...

This valuable study presents a mouse gastruloid model that can be used to generate hematopoietic progenitors as well as leukemic cells. However, in its current form, the manuscript is inadequate ...

Australian cancer researchers are the first to establish a next-generation gene-editing tool for modeling and interrogating human disease.

Two new compounds may be able to treat retinitis pigmentosa, a group of inherited eye diseases that cause blindness. The compounds were identified using a virtual screening approach.

Findings indicate that altering glucose metabolism in T cells boosts their therapeutic potential against melanoma, paving the way for improved immunotherapies.

How cells respond to mitochondrial DNA (mtDNA) damage is not fully understood. This study uncovers a mechanism of mtDNA ...

NanoCas, an ultracompact nuclease, enables efficient muscle editing in NHPs using a single AAV - advancing gene editing for hard-to-target tissues.

Scientists have discovered a potential new treatment for familial hypercholesterolemia, inspired by signs seen in the Mona ...