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  2. Exagamglogene autotemcel - Wikipedia

    en.wikipedia.org/wiki/Exagamglogene_autotemcel

    The treatment was approved in the United Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023. [10] [11] [12] It was approved in the United States for the treatment of sickle cell disease in December 2023 and for the treatment of transfusion-dependent beta thalassemia in January 2024. [13 ...

  3. Lovotibeglogene autotemcel - Wikipedia

    en.wikipedia.org/wiki/Lovotibeglogene_autotemcel

    Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1] [3] [4] [5]The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with ...

  4. Sickle cell disease - Wikipedia

    en.wikipedia.org/wiki/Sickle_cell_disease

    Sickle cell disease (SCD), also simply called sickle cell, is a group of hemoglobin-related blood disorders that are typically inherited. [2] The most common type is known as sickle cell anemia. [2] Sickle cell anemia results in an abnormality in the oxygen-carrying protein haemoglobin found in red blood cells. [2]

  5. FDA advisers review sickle cell treatment that could cure the ...

    www.aol.com/news/fda-advisers-review-sickle-cell...

    The FDA is reviewing a drug that could cure sickle cell anemia. Right now bone marrow or stem cell transplants are the only FDA-approved cures. FDA advisers review sickle cell treatment that could ...

  6. US FDA approves two gene therapies for sickle cell disease - AOL

    www.aol.com/news/us-approves-two-gene-therapies...

    (Reuters) -The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel ...

  7. Two gene therapies for sickle cell disease approved in US - AOL

    www.aol.com/news/two-gene-therapies-sickle-cell...

    Regulators on Friday approved two gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the U.S. The Food and ...

  8. Allele-specific oligonucleotide - Wikipedia

    en.wikipedia.org/wiki/Allele-specific_oligo...

    The human disease sickle cell anemia is caused by a genetic mutation in the codon for the sixth amino acid of the blood protein beta-hemoglobin.The normal DNA sequence G-A-G codes for the amino acid glutamate, while the mutation changes the middle adenine to a thymine, leading to the sequence G-T-G (G-U-G in the mRNA).

  9. Victoria Gray - Wikipedia

    en.wikipedia.org/wiki/Victoria_Gray

    Victoria Gray was the first patient ever to be treated with the gene-editing tool CRISPR for sickle-cell disease. [1]This marked the initial indication that a cure is attainable for individuals born with sickle-cell disease and another severe blood disorder, beta-thalassemia.

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