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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.
news & eventsSickle cell disease cure in sights of UAB Stem Cell InstituteTim Townes has a dream. He has a dream t...
education & researchImmunogenicity and Safety of 10-valent Pneumococcal Nontypeable Haemophilus influenzae Protein D Conjugate Vaccine (...BACKGROUND: Immunogenicity, safety and r...
news & eventsA multiple drug approach to preventing sickle cell crisisSickle cell disease is characterized by ...
education & researchEffect of crizanlizumab on pain crises in subgroups of patients with sickle cell disease: A SUSTAIN study analysisThe cell adhesion molecule P-selectin pl...
videos & visualsCrizanlizumab for the Prevention of Pain Crises in Sickle Cell Diseasehttps://www.youtube.com/watch?v=CWTW5pDP...
videos & visualsFacebook Live: Stem Cells and Sickle Cell Diseasehttps://www.youtube.com/watch?v=IPzNx8i_...
videos & visualsConstance Benson Cured of Sickle Cell, Speaking at Sickle Cell Disease & Gene Editing Briefing in DChttps://www.youtube.com/watch?v=ROtPSLJl...
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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.