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Stem cell gene-editing method may be breakthrough for sickle cell research
Researchers have cured mice with a genetic blood disease by correcting mutated genes in blood cell-producing stem cells. The method offers a new therapeutic approach for treating blood disorders, such as sickle cell disease and thalassemia.
The gene-editing technique can be administered easily into living animals and notably decreases off-target effects (side effects), unlike the CRISPR technique, which displays problems in those regards.
The study, “In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery,” was published in Nature Communications.
education & researchAddressing challenges of clinical trials in acute pain: The Pain Management of Vaso-occlusive Crisis in Children and...BACKGROUND/AIMS: Neuropathic pain is a k...
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Community CenterFamily Celebrates 7-Year-Old’s Sickle Cell Cure Thanks to Sister’s Life-Saving GiftOne 7-year-old who suffered his whole li...
news & eventsbluebird bio Announces Publication of Case Study on First Patient with Severe Sickle Cell Disease Treated with Gene ...Patient treated with LentiGlobinTM drug ...
Community CenterToday’s Faces of Sickle Cell Disease: John Tisdale, MDFor many years, John Tisdale has been wo...
news & eventsVitamin D Supplements May Reduce Pain-related Emergency Room Visits in Children With SCDTaking vitamin D supplements may lower t...
news & eventsFDA Advisory Committee Recommends Approval of Endari™ from Emmaus Life Sciences for the Treatment of Sickle Cell D...Emmaus Life Sciences Inc. announced toda...
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.