News Medical on MSN13h
Using CRISPR to remove extra chromosomes in Down syndrome
Gene editing techniques may eventually allow trisomy to be treated at the cellular level, according to an in vitro ...
News Medical on MSN20h
Gene therapy breakthrough offers hope for severe Dravet syndrome cases
AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...
Rare genetic syndrome affects male and female brains in distinct ways
Researchers at Tel Aviv University, led by Prof. Illana Gozes, examined the effects of different mutations in the ADNP protein, which is essential for normal brain development and aging, on the brain ...
His daughter has an ultra-rare genetic disorder. His push to treat it just hit a major milestone
A clinical trial by Shionogi is a milestone in the search to treat Jordan's Syndrome, a rare genetic disorder named after a ...