Celia Grace Hamlett was 4-years-old when she traveled with her parents from Alabama to Minneapolis to receive life-saving treatment for a rare genetic disease, Metachromatic Leukodystrophy or MLD.

Penn and CHOP researchers will utilize the new protocol to distribute customizable drugs that target specific genetic disorders.

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...

COLUMBUS, OHIO, UNITED STATES, January 3, 2024 /EINPresswire.com/ -- Planet TV Studios, a prominent producer of groundbreaking television series, proudly announces ...

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol ...

Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...