Signal
19 articles covering "gene-editing"
Shanghai Jiao Tong University is investigating neuroscientist Zilong Qiu after a 6-year-old girl died from a severe immune reaction in an experimental gene-editing trial that went undisclosed.
Jennifer Doudna's team used AI protein design to create synthetic TnpB nucleases that edited human genes more efficiently than the natural enzyme.
The Jennifer Doudna-founded CRISPR biotech filed to go public under ticker SCTX as its lead cholesterol therapy remains early in human testing.
ARPA-H's new THRIVE program is funding seven teams to build reusable gene-editing platforms for rare pediatric diseases, aiming to move beyond one-patient-at-a-time therapies like the one made for infant KJ Muldoon.
A single infusion of VERVE-102 reduced LDL-C by up to 62% and PCSK9 by up to 88% in a 35-patient trial, with effects sustained up to 18 months.
A University of Utah-led international team reports a new CRISPR nuclease, Cas12a2, that uses RNA guides to trigger widespread DNA shredding and cell death specifically in diseased cells such as KRAS-mutant cancers and HPV-infected cells, while leaving healthy cells unharmed.
First-quarter results from both partners show CASGEVY past 500 treatment starts, a US filing for ages 5 to under 12, and CRISPR Therapeutics' in vivo pipeline advancing across four liver-directed programs.
Eli Lilly's deal with Bezos-backed Profluent funds AI-designed recombinases that aim to insert entire genes, going beyond CRISPR's localized cuts.
Two companion Nature Biotechnology papers map over 30,000 CRISPR edits across sorghum's regulatory DNA, achieving unprecedented control over photosynthesis genes with implications for crop yields and carbon capture.
UT Austin and Metagenomi researchers engineered Al3Cas12f RKK, a compact CRISPR enzyme that achieves over 80% editing efficiency while fitting inside AAV delivery vehicles.
Prime Medicine is pursuing accelerated approval for PM359, an autologous prime editing therapy that restored immune function in both patients treated for chronic granulomatous disease, marking the first clinical use of prime editing technology.
A two-particle gene-editing system published in Nature creates functional CAR-T cells in vivo, eliminating the costly weeks-long manufacturing process and clearing leukemia, myeloma, and solid tumors in preclinical models.