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LATEST NEWS
No, CRISPR Hasn't Cured Down Syndrome. Here's What the Viral Study Really Showed
A viral post claims scientists can now remove the extra chromosome behind Down syndrome. The 2025 Mie University study is real, but it was done in lab-grown cells, not babies, and it is far from a treatment. Here is the myth vs. fact.
13 hours ago4 min read
One CRISPR Infusion Kept Cholesterol and Triglycerides Down for a Full Year
One-year data for CTX310, a single-infusion CRISPR therapy that switches off ANGPTL3 in the liver, showed LDL cholesterol down 53% and triglycerides down 48% at the top dose, with no treatment-related serious side effects.
13 hours ago4 min read
Beacon's Gene Therapy for X-Linked Retinitis Pigmentosa Succeeds in a Pivotal Trial
Laru-zova met its primary goal in the pivotal VISTA trial: 31% of high-dose patients gained three or more lines of dim-light vision, versus none in the control group. It is the first XLRP gene therapy to win a late-stage trial.
2 days ago4 min read
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GENE TECH TIMES
The Future Has Arrived
Lenmeldy: The First FDA-Approved Gene Therapy for Metachromatic Leukodystrophy in Children
Lenmeldy, a groundbreaking gene therapy developed by Orchard Therapeutics, has recently received FDA approval, making it the first and only treatment available for children with Metachromatic Leukodystrophy (MLD), a rare genetic disorder that severely affects the brain. Metachromatic Leukodystrophy is a rare lysosomal storage disorder caused by mutations in the ARSA and/or PSAP genes. These mutations result in the accumulation of sulfatide, leading to the degeneration of esse
Apr 15, 2024
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