Tagged: Gene therapy

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Relief for retinal neurons under pressure

Advancing age predisposes us to a number of neurodegenerative diseases, yet the underlying mechanisms are poorly understood. With some 70 million individuals affected, glaucoma is the world’s leading cause of irreversible blindness. Glaucoma is...

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First Data from Anti-Aging Gene Therapy

Last year, Elizabeth Parrish, the CEO of Seattle-based biotech firm BioViva, hopped a plane to Colombia, where she received multiple injections of two experimental gene therapies her company had developed. One is intended to...

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Junk DNA Helps Suppress Breast Cancer

A piece of noncoding DNA helps prevents cells from turning cancerous, and does so via two mechanisms. The noncoding DNA, which is called GNG12-ASI, acts both directly by regulating the transcription of a cell-replication...

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Building connections by gene therapy

Voluntary movement requires a molecular conversation between nerves and muscles. This conversation occurs at the neuromuscular junction, a structure where nerves and muscle physically connect. People with diseases characterized by muscle weakness have aberrantly...

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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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Genome Editing Cuts Out HIV

Like other retroviruses, the genetic material of HIV wedges itself into the genome of its human host. While antiretroviral therapies are effective at repressing HIV, they don’t eliminate the integrated virus, which can lie...

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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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