Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Lucas Oon has Angelman syndrome, which is characterised by a happy demeanour and an easily excitable nature. Read more at ...

UBE3A is an E3 ubiquitin ligase encoded by an imprinted gene whose maternal deletion or duplication leads to distinct neurodevelopment disorders Angelman and Dup15q syndromes. Despite the known ...

SINGAPORE – Laughter fills the consultation room whenever Lucas Oon, 17, is with the team managing his care – pediatric neurologist Jeremy Lin, assistant nurse clinician Kimberly Quek and ...

Flyers showing the names, pictures, and phone numbers of Immigration and Customs Enforcement (ICE) agents have surfaced in a Southern California neighborhood. Multiple federal law enforcement ...

Having two copies of the MTHFR gene mutation may raise your risk for certain health conditions, though more research is needed. You may have the mutation and not find out unless you undergo ...

(AP Photo/Gene J. Puskar) The Pittsburgh Penguins pay tribute to Penguins Hockey Hall of Fame announcer Mike Lange before an NHL hockey game between the Pittsburgh Penguins and the Washington Capitals ...

When the monkeys were single-celled embryos, scientists had used CRISPR editing tools to silence, or “knock out”, a gene that helps regulate the body’s internal clock. Its disruption is ...

Building a gene editor took months. With CRISPR even high-schoolers can get hold of editing systems in the time it takes to order RNA sequences online and have them shipped by FedEx. In a short ...