Researchers at the Hebrew University of Jerusalem, Israel, have attempted to restore color vision to completely color-blind subjects using gene therapy. In their paper, "Seeing color following gene augmentation therapy in ...
Hematopoietic stem cells (HSCs) are rare cells found in the bone marrow that produce red blood cells, white blood cells, and platelets. Their correct functioning is indispensable for the growth and health of an organism. ...
Jul 10, 2023
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The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A, a life-threatening hereditary bleeding disorder.
Jul 3, 2023
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Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia. These two common life-threatening anemias afflict millions of individuals across the globe.
Jun 29, 2023
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An international team of researchers has developed a new method to deliver drugs into the inner ear. The discovery was made possible by harnessing the natural flow of fluids in the brain and employing a little understood ...
Jun 28, 2023
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Prof. Yossi Buganim and his research team at the Faculty of Medicine at the Hebrew University of Jerusalem have achieved a groundbreaking milestone in the field of cell reprogramming. Their latest study, published in Nature ...
Jun 27, 2023
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Macquarie Medical School researchers have developed a technique for a gene therapy that could help treat the world's leading cause of irreversible blindness. The findings are published in the journal Molecular Therapy.
Jun 27, 2023
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The U.S. Food and Drug Administration on Thursday approved the drug Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD).
Jun 26, 2023
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An innovative gene therapy may help treat a severe and fatal developmental epilepsy syndrome that affects children. Researchers hope that this genetic therapy can also be adapted to other types of genetic epilepsies and think ...
Jun 22, 2023
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It's not easy to distinguish between the dozens of subtypes of limb girdle muscular dystrophy—a rare, genetic muscle disease characterized by weakness in the hips and shoulders that causes difficulty walking and lifting ...
Jun 15, 2023
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