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AIDS cure may have two main pathways: experts (Update)

Investigators are looking into two main paths toward a cure for AIDS, based on the stunning stories of a small group of people around the world who have been able to overcome the disease.

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Cancer study overturns current thinking about gene activation

(Medical Xpress)—A new Australian study led by Professor Susan Clark from Sydney's Garvan Institute of Medical Research shows that large regions of the genome – amounting to roughly 2% – are...

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Altering eye cells may one day restore vision

(Medical Xpress)—Doctors may one day treat some forms of blindness by altering the genetic program of the light-sensing cells of the eye, according to scientists at Washington University School of...

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Myth that UK supply of innovative new pharma drugs is drying up

The widely held belief that the UK supply of innovative new medicines has conspicuously dwindled in recent decades, is not borne out by the evidence, reveals research published in the online journal...

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Novel intercellular transportation system may have potential for delivering...

(Medical Xpress)—Important new research from UMass Medical School demonstrates how exosomes shuttle proteins from neurons to muscle cells where they take part in critical signaling mechanisms, an...

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Discovery shows fat triggers rheumatoid arthritis

Scientists have discovered that fat cells in the knee secrete a protein linked to arthritis, a finding that paves the way for new gene therapies that could offer relief and mobility to millions worldwide.

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Over-produced autism gene alters synapses, affects learning and behavior in mice

A gene linked to autism spectrum disorders that was manipulated in two lines of transgenic mice produced mature adults with irreversible deficits affecting either learning or social interaction.

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Gene therapy restores immunity in children and young adults with rare...

Gene therapy can safely rebuild the immune systems of older children and young adults with X-linked severe combined immunodeficiency (SCID-X1), a rare inherited disorder that primarily affects males,...

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Early gene therapy results in Wiskott-Aldrich syndrome promising

Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune...

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Shrinking tumors with an RNA triple-helix hydrogel glue

Twenty years ago, scientists discovered that short strands of RNA known as microRNA help cells to fine-tune their gene expression. Disruption or loss of some microRNAs has been linked to cancer,...

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Researchers use gene therapy to extend estrogen's protective effects on memory

The hormone estrogen helps protect memory and promote a healthy brain, but this effect wanes as women age, and even estrogen replacement therapy stops working in humans after age 65. Now researchers at...

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Suicide gene therapy kills prostate tumor cells

Results from a long-term clinical trial conducted by cancer researchers at Houston Methodist Hospital show that combining radiation treatment with "suicide gene therapy," a technique in which prostate...

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Physicists devise gene therapy platform for macular degeneration patients

Millions of adults over age 50 struggle each year with vision loss caused by damage to the retina or common macular degeneration.

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CRISPR treats genetic disorder in adult mammal

Researchers have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy. This marks the first time that CRISPR has successfully treated a genetic disease inside a fully developed...

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Proposed link between liver cancer and adeno-associated virus challenged in...

The conclusion drawn from a recent study that insertion of adeno-associated virus 2 (AAV2) into human DNA causes mutations leading to the development of hepatocellular carcinoma (HCC) was resoundingly...

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Researchers bring gene editing to patients with deadly diseases

Tiny vials of recently repaired blood cells are thriving in a Stanford incubator, proof that a powerful new gene-editing technique is fixing errant genes that cause so much human suffering.

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Scientists uncover how part of a protein helps primates fight HIV

Scientists have uncovered part of a protein found in humans and other primates that can help us fight off HIV. In a new study published in the journal Heliyon, researchers discover how this structure...

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Drug 'cocktail' could restore vision in optic nerve injury

Research from Boston Children's Hospital suggests the possibility of restoring at least some visual function in people blinded by optic nerve damage from glaucoma, estimated to affect more than 4...

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Gene therapy for rare bleeding disorder achieves proof-of-concept

Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a...

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Edited stem cells offer hope of precision therapy for blindness

Using a new technology for repairing disease genes—the much-talked-about CRISPR/Cas9 gene editing—University of Iowa researchers working together with Columbia University Medical Center...

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