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网页5 天之前 · One sickle cell disease (SCD) patient may need as many as 50 units of blood and up to 25 units of platelets for the new cell-based gene therapies such as Casgevy and Lyfgenia to be successfully administered, according to the American Red Cross. As a part of…. September 16, 2024 Columns by Dunstan Nicol-Wilson.
FDA approves Casgevy, 1st gene-editing therapy to treat sickle cell
网页2023年12月8日 · The U.S. Food and Drug Administration (FDA) has approved the gene-editing therapy exagamglogene autotemcel (exa-cel) — now known as Casgevy — for treating sickle cell disease (SCD).. The therapy from Vertex Pharmaceuticals and CRISPR Therapeutics is indicated for patients ages 12 and older who have recurrent vaso-occlusive crises (VOCs). With the decision, Casgevy has become the first cell ...
Top 10 sickle cell disease stories of 2023
网页2024年1月2日 · No. 7 – Fewer sickle-shaped red blood cells with gene therapy. The experimental gene therapy BCH-BB694 reduced the number of red blood cells that produce faulty hemoglobin S, the hallmark of SCD. The therapy also curbed the tendency of these cells to take on a sickle-shaped form, as revealed …
1st SCD Trial Patient Shows CTX001 Gene Editing to be Safe, …
网页2019年11月20日 · by Marta Figueiredo, PhD November 20, 2019. CTX001 safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crises in the first severe sickle cell disease (SCD) patient receiving the therapy, according to preliminary data from a Phase 1/2 clinical trial. CTX001 is a CRISPR-based gene editing therapy developed ...
Risk of blood clots more likely with sickle cell trait, but still low
网页3 天之前 · Still, the researchers said, the risk of these blood clots was low, occurring in just over 2% of people carrying SCT. The study, “ Ancestry-Independent Risk of Venous Thromboembolism in Individuals with Sickle Cell Trait vs. Factor V Leiden,” was published in Blood Advances. “I hope this study informs health professionals, particularly hematologists, that when thinking about risks ...
Most sickle cell patients VOE-free after Lyfgenia gene therapy
网页2023年12月12日 · Most sickle cell disease (SCD) patients treated with the gene therapy Lyfgenia (lovotibeglogene autotemcel) in clinical trials were free from vaso-occlusive events (VOEs) after a median of three years of follow-up. That’s according to new data from Lyfgenia’s developer, Bluebird Bio, at the 65th American …
Top 10 Sickle Cell Disease Stories of 2022
网页2022年12月29日 · Here we’ve compiled a list of the 10 most-read articles published on our site in 2022, with a brief description of each. No. 10 — “ Trial of Cell Therapy to Raise HbF Levels Showing Potential, But Sanofi Leaving ”. SAR445136 is a gene-edited cell therapy designed to boost the production of fetal hemoglobin (HbF) in a patient’s blood ...
Sickle Cell Patient Becomes World's First to be Successfully...
网页by Magdalena Kegel March 7, 2017. A 13-year-old boy with sickle cell disease has become the world’s first patient to be successfully treated with gene therapy. The unidentified teenager remains free from sickle crises and other signs of the disease 15 months after his treatment, which was conducted as part of a Phase 1/2 clinical trial ...
Gene Therapy - Sickle Cell Disease News
网页Gene therapy is an experimental technique that aims to treat genetic diseases by altering a disease-causing gene or introducing a healthy copy of a mutated gene to the body. The U.S. Food and Drug Administration approved the first gene therapy for an inherited disease — a genetic form of blindness — in December 2017. Gene therapy for sickle cell anemia
Non-viral Gene Editing of Marrow Stem Cells Shows Potential for …
网页by Steve Bryson, PhD May 11, 2021. A non-viral, CRISPR-based gene-editing tool showed a potential to reverse mutations that cause sickle cell disease (SCD) by successfully editing genes in stem cells within the bone marrow of mice. Data from this proof-of-concept study also found that changes in stem cell genes were sustained for more than one ...
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