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.
The Times of Israel on MSN
In world first, Israeli scientists use RNA-based gene therapy to stop ALS deterioration
By adding a microRNA molecule to ALS models, Tel Aviv University, Ben-Gurion University and Weizmann Institute researchers ...
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 ...
Penn and CHOP researchers will utilize the new protocol to distribute customizable drugs that target specific genetic disorders.
7don MSN
First-in-human trial of CRISPR gene-editing therapy safely lowers cholesterol and triglycerides
In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL 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 ...
Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback