Scientists have created lipid nanoparticles that can deliver mRNA to the retina, offering an opportunity for the development of additional gene therapies for the eye. Studies...
Ten children with a rare condition of severe combined immunodeficiency with Artemis deficiency had their immune systems partially or completely restored by gene replacement therapy health...
Sickle cell disease is a highly debilitating condition that affects up to 40% of the African population and patients suffer from intolerable pain, organ damage, and...
Ashland, Kentucky (WSAZ)-Pancreatic cancer has the highest mortality rate of all major cancers, but breakthrough treatments offer new hope. “I never expected to die of pancreatic...
An international research team led by scientists at the University of California, San Diego Medical School, found that genetic therapy that inhibits signaling in target neurons...
Vision of a man who lost sight because of Retinitis pigmentosa (RP) was partially recovered by optogenetic treatment in the first reported case of functional recovery...
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