The blood-brain barrier (BBB) is an imposing foe for gene therapy. Formed of cells wedged tightly together, the BBB keeps toxins and pathogens that may be present in the blood from entering brain tissue, but it also keeps ...
Oct 10, 2022
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Idiopathic pulmonary fibrosis could become the first degenerative disease to be treated through the science of aging.
Oct 6, 2022
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New educational materials to help the sickle cell disease community learn about gene therapies for the disease are now available from researchers at the National Human Genome Research Institute (NHGRI), part of the National ...
Sep 28, 2022
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In a new paper, University of California, Irvine researchers explain how precision genome editing agents have enabled precise gene correction and disease rescue in inherited retinal diseases (IRDs). The study, titled, "Precision ...
Sep 23, 2022
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Scientists at St. Jude Children's Research Hospital have created a tool that can find safe places to introduce genes into human DNA. The tool is an early step in the process to improve the safety and efficacy of gene and ...
Sep 22, 2022
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The oncogene EVI1 causes an aggressive type of leukemia, but its exact function has been a mystery. A research team led by scientists from Vienna now showed that EVI1's cancer causing effect relies on activating a single ...
Sep 20, 2022
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Bluebird Bio Inc.'s gene therapy for a brain-wasting disease received accelerated approval from U.S. regulators, making it the first treatment of its kind for children living with cerebral adrenoleukodystrophy.
Sep 19, 2022
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Alveolar rhabdomyosarcoma is a type of rare pediatric tumor. For more than 40 years there have not been any new developments regarding treatment. Research led by Prof. Dr. Anton Henssen at Charité University Berlin has now ...
Sep 19, 2022
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Blocking the inhibition of PKD1 and PKD2 gene expression by deleting a binding site for microRNAs hindered the formation and growth of kidney cysts in autosomal dominant polycystic kidney disease (ADPKD) models, UT Southwestern ...
Sep 16, 2022
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Researchers from the National Eye Institute (NEI) have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early ...
Sep 8, 2022
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