Laura Covill from the Hematology Unit/HERM, at the Department of Medicine, Huddinge (MedH), is defending her thesis titled "Genetic and epigenetic diagnostics of viral susceptibility and inborn errors of immunity," on 29 ...
Nov 26, 2024
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Efforts to develop a gene therapy for Diamond-Blackfan anemia (DBA)—a rare, life-threatening disorder in which bone marrow cannot make mature, functioning red blood cells—have been hampered by the fact that at least 30 ...
Nov 26, 2024
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The United States Food and Drug Administration has just approved the first-ever clinical trial that uses CRISPR-Cas13 RNA editing. Its aim is to treat an eye disease called wet age-related macular degeneration that causes ...
Nov 23, 2024
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A study from Tel Aviv University expands the understanding of the biological mechanism underlying genetically-based autism, specifically mutations in the SHANK3 gene, responsible for nearly one million cases of autism worldwide. ...
Nov 20, 2024
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Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene Therapy. To improve the efficacy of gene therapy when using anti-sickling beta globin gene transfer, ...
Nov 19, 2024
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Research that was carried out by Baylor College of Medicine and Texas Children's Hospital doctors has led to the U.S. Food and Drug Administration (FDA) granting Orphan Drug Designation to a treatment for developmental and ...
Nov 15, 2024
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University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success using a gene therapy treatment in an animal model. The approach, which uses CRISPR-Cas9 genome editing ...
Nov 12, 2024
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Diamond-Blackfan anemia (DBA) is a potentially life-threatening condition that can cause severe anemia and other abnormalities. It's a rare genetic blood disorder that occurs when the bone marrow fails to make red blood cells.
Nov 11, 2024
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In a new study, a team of researchers, including Anneliene Jonker, has published, in Nature Reviews Drug Discovery, the first roadmap for developing genetic therapies for ultra-rare diseases. These conditions affect only ...
Nov 6, 2024
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IgA nephropathy is an autoimmune kidney disease, and complement, a component of the innate immune system, plays a role in the condition's pathogenesis. Investigators have developed and tested a novel gene therapy that enters ...
Oct 28, 2024
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