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1. www.nature.com › articles › d41586/021/02138-wGene therapies close in on a cure for sickle-cell disease

   www.nature.com › articles › d41586/021/02138-w

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   Aug 25, 2021 · Sickle-cell disease presents a near-ideal opportunity to tap the power of gene therapy because the disorder typically arises from a mutation in a single nucleotide in one gene. That gene encodes ...

2. www.nhlbi.nih.gov › news › 2023NIH statement on new FDA-approved gene therapies for sickle ...

   www.nhlbi.nih.gov › news › 2023

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   Dec 8, 2023 · One of the new sickle cell treatments uses the CRISPR gene-editing system, a first for humans in the U.S. NIH funds the Cure Sickle Cell Initiative to help speed the development of cures for sickle cell disease, which takes advantage of the latest genetic discoveries and technological advances to move the most promising genetic-based curative ...

3. my.clevelandclinic.org › health › diseasesSickle Cell Disease (SCD): Types, Symptoms & Causes

   my.clevelandclinic.org › health › diseases

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   Aug 3, 2023 · Sickle cell disease treatment includes medications, transfusions, blood and marrow transplant and gene therapy. Sickle cell disease treatment may begin with antibiotics . Newborns with severe SCD will receive antibiotics twice a day until they’re 5 years old to prevent infection.

4. www.hopkinsmedicine.org › news › newsroomA Cure for Sickle Cell Disease | Johns Hopkins Medicine

   www.hopkinsmedicine.org › news › newsroom

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   Dec 12, 2023 · A high-dose regimen, known as myeloablative conditioning, with a fully matched bone marrow transplant has been known to cure sickle cell disease for decades. However, its use has been limited — particularly in older patients with organ damage caused by sickle cell disease — because of concerns about side effects, says Brodsky.

5. www.nytimes.com › sickle-cell-fda-cure-crisprPanel Tells F.D.A. That CRISPR Sickle Cell Cure Is Safe ...

   www.nytimes.com › sickle-cell-fda-cure-crispr

   Oct 31, 2023 · Oct. 31, 2023. A panel of experts said on Tuesday that a groundbreaking treatment for sickle cell disease was safe enough for clinical use, setting the stage for likely federal approval by Dec. 8 ...

6. www.yalemedicine.org › news › gene-therapies-sickleCasgevy and Lyfgenia: Two Gene Therapies Approved for Sickle ...

   www.yalemedicine.org › news › gene-therapies-sickle

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   Dec 19, 2023 · Both treatments—Casgevy™, which is made by Vertex Pharmaceuticals and CRISPR Therapeutics, and Lyfgenia™, by Bluebird Bio—are for people 12 and older. Sickle cell disease is a red blood cell disorder that affects hemoglobin, the protein that carries oxygen throughout the body. These two therapies work in different ways, but both are ...

7. hospital.uillinois.edu › primary-and-specialtyCure for Sickle Cell Disease in Chicago | UI Health

   hospital.uillinois.edu › primary-and-specialty

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   Stem cell transplant in Chicago cured Julius and Desmond Means from Sickle Cell Disease. The following year, brothers Julius and Desmond Means were cured of sickle at UI Health following a stem cell transplantation from their older brother, Clifford. For the sibling to be a compatible donor, at least eight of the 10 known human leukoute antigen ...