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

With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery ...

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 ...

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. | ...

Neuroblastoma is a solid tumor that occurs in children. When high-risk, the disease has a poor prognosis. Decades ago, adding ...

Down syndrome, or trisomy 21, is caused by the presence of a third copy of the 21st chromosome. Affected individuals have ...

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 ...

The authors have demonstrated the use of adenine base editors delivered via adeno-associated viruses to introduce edits in the mitochondrial genome. The manuscript describes the methodology well, and ...

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