When the monkeys were single-celled embryos, scientists had used CRISPR editing tools to silence, or “knock out”, a gene that ...
Colossal Biosciences leaders say the fluffy, golden-haired mice help validate their technique to “de-extinct” species, but ...
CRISPR-based Spotlight Therapeutics has shut down, former CEO Mary Haak-Frendscho, Ph.D., confirmed with Fierce Biotech. | CRISPR-based Spotlight Therapeutics has shut down, former CEO Mary ...
With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery ...
A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.
Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...
Down syndrome, or trisomy 21, is caused by the presence of a third copy of the 21st chromosome. Affected individuals have ...
Neuroblastoma is a solid tumor that occurs in children. When high-risk, the disease has a poor prognosis. Decades ago, adding ...
Regeneron’s gene therapy, dubbed DB-OTO, is a cell-selective AAV gene therapy for children with hearing loss stemming from a ...
Gene regulation underpins nearly every biological process—from cell development to responses to environmental changes, and ...