Category: Gene Therapy

Oligonucleotide Therapeutics Near Approval

Cold Spring Harbor Laboratory molecular geneticist Adrian Krainer was at a National Institutes of Health workshop in 1999 when he first learned about the crippling neurodegenerative disease spinal muscular atrophy (SMA)—the leading genetic cause...

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Mitochondria Swap

Scientists have used two methods to generate patient-specific pluripotent stem cells with normal mitochondria for people with defects in these organelles, according to a study published today (July 15) in Nature. The first method...

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CRISPR corrects β-thalassaemia

A common genetic blood disorder has been corrected in cultured stem cells by using a cutting-edge genome-editing technique. The disorder β-thalassaemia is characterized by reduced levels of haemoglobin due to mutations in the gene...

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Gene therapy creates biological pacemaker

Gene therapy could soon join the electronic pacemaker as a treatment for a weak heart. By inserting a specific gene into heart-muscle cells, researchers can restore a normal heart rate in pigs at least...

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Gene Therapy Improves Sight

A few patients with a rare degenerative eye disease, called choroideremia, had partially restored vision following a gene therapy trial to deliver a healthy copy of the gene CHM to the eye. The preliminary trial, published...

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