Monday's Daily Dose with Monica Robins: Gene therapy breakthrough, new cholesterol drug and stroke risk linked to alcohol

From revolutionary CRISPR treatment to heavy drinking dangers, here's what you need to know about the latest health research.

Gene therapy for sickle cell and β-thalassemia works by disrupting three-dimensional genome structure

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...
MedPage Today on MSN

Gene Therapy Shows Promise for Cutting LDL in Half in Tough-to-Treat Dyslipidemia

N EW ORLEANS – A single injection of a gene-edited protein was deemed productive and safe in patients with dyslipidemia that ...
Futurism on MSN

Scientists Say They’ve Figured Out a Way to Reprogram the Pancreas to Produce GLP-1s Without Ozempic

Scientists say they've come up with a way for the body to produce its own store of glucagon-like peptide-1 (GLP-1).
Fierce Biotech

uniQure's ballyhooed gene therapy for Huntington's hits FDA roadblock

The FDA is said to be making a U-turn on uniQure’s one-time gene therapy candidate for Huntington’s disease despite the ...

A Gene Editing Therapy Cut Cholesterol Levels by Half

An experimental gene-editing therapy developed by Crispr Therapeutics is showing promise for treating heart disease.
BioSpace

Patient Dies After Receiving Intellia’s CRISPR Gene Therapy Candidate

The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred. Intellia is working with experts to create a risk management program for nex ...
EurekAlert!

RNA-based gene therapy may enable effective treatment for ALS – a fatal incurable neurodegenerative disease

Researchers at Tel Aviv University led a large-scale international study that opens a new avenue for treating the fatal ...

New gene therapy approach could reverse ALS, say scientists

"Our finding is important because it provides a fresh understanding of how ALS begins and progresses, and it opens the door to a potential new treatment strategy: restoring this lost RNA signal ...
Ophthalmology Times

Opus Genetics has Type B RMAT meeting with FDA for its gene therapy candidate, OPGx-LCA5

Opus Genetics has completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the US Food and Drug Administration (FDA) regarding OPGx-LCA5, its gene therapy candidate for Leber ...