A team of researchers has developed an inhalable vaccine that successfully protects against the COVID virus. It also opens the door to delivering other messenger RNA (mRNA) therapeutics for gene replacement therapy and other ...
Aug 20, 2023
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Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.
Nov 18, 2021
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An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National ...
May 12, 2021
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In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA). Approval included all children with ...
Aug 25, 2020
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Preliminary data from an expanded access study of an investigational gene therapy in two patients with infantile Tay-Sachs disease indicates the potential to modify the rate of disease progression, according to an upcoming ...
Oct 24, 2019
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Advances in preclinical research are now being translated into innovative clinical solutions for blindness, a review published in the 10th Anniversary Series of Science Translational Medicine reports.
Jun 5, 2019
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A trial for a new gene therapy, known as AXO-Lenti-PD, aimed at improving the supply of dopamine in the brains of people with Parkinson's disease has been launched by researchers at UCL and University College London Hospitals ...
Oct 25, 2018
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A team at the University of Manchester have developed a novel stem cell gene therapy approach to treat children with a devastating genetic disease. The approach is currently being developed for clinical trial in patients ...
Jul 6, 2018
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A team of Canadian physicians and researchers is believed to be the first in the world to have used gene therapy to treat a patient with Fabry disease, a rare inherited enzyme deficiency that can damage major organs and shorten ...
Feb 16, 2017
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After decades investigating a rare, life-threatening condition that cripples the muscles, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently ...
Jan 26, 2017
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