When the monkeys were single-celled embryos, scientists had used CRISPR editing tools to silence, or “knock out”, a gene that ...

Gene editing techniques may eventually allow for trisomy disorders to be treated at the cellular level, according to an in ...

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.

Gene editing techniques may eventually allow trisomy to be treated at the cellular level, according to an in vitro ...

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 ...

When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters ...

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...

Regeneron’s gene therapy, dubbed DB-OTO, is a cell-selective AAV gene therapy for children with hearing loss stemming from a ...

Decades ago, researchers showed that Down syndrome (also known as trisomy 21), was caused when individuals carried an extra ...

Gene regulation underpins nearly every biological process—from cell development to responses to environmental changes, and ...