Gene therapy targeting BCL11A to induce fetal hemoglobin and reduce sickle hemoglobin in patients with Sickle Cell Disease

靶向 BCL11A 的基因疗法可诱导胎儿血红蛋白并降低镰状细胞病患者的镰状血红蛋白

• 批准号: 10083551
• 负责人: DAVID A WILLIAMS
• 金额: $ 120.04万
• 依托单位: BOSTON CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-07-22 至 2024-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10083551
• 关键词: Adult; Allogenic; Architecture; Attenuated; Autologous; Biocompatible Materials; Biological; Cells; Clinical; Data; Disease; Engraftment; Enrollment; Erythrocytes; Erythroid; Erythroid Cells; Feasibility Studies; Fetal Hemoglobin; Fetal Reduction; Future; Gene Transfer; Gene-Modified; Genes; Globin; Goals; Half-Life; Health; Hematology; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Hemoglobin; Hemoglobinopathies; Human; Incidence; Individual; Laboratories; Lead; Life; MicroRNAs; Molecular; Outcome; Patients; Phenotype; Risk; Severities; Siblings; Sickle Cell; Sickle Cell Anemia; Sickle Hemoglobin; Transgenes; Transplantation; base; beta Thalassemia; clinical effect; cohort; curative treatments; disease phenotype; fetal; gamma Globin; gene therapy; gene transfer vector; graft failure; graft vs host disease; improved; in vivo; knock-down; mathematical model; mortality; mouse model; mutant; phase I trial; polymerization; pre-clinical; prevent; safety and feasibility; sickling; small hairpin RNA; stem cells; targeted treatment; vector

No abstract provided

未提供摘要

项目成果

Magnetic resonance imaging metrics of oxygen extraction fraction: Contradictions or insight into pathophysiological mechanisms?

• DOI: 10.1002/ajh.26567
• 发表时间: 2022-06-01
• 期刊: AMERICAN JOURNAL OF HEMATOLOGY
• 影响因子: 12.8
• 作者: Stout, Jeffrey N.;Lin, Pei-Yi;Sutin, Jason;Higgins, John;Grant, P. Ellen
• 通讯作者: Grant, P. Ellen

Corrigendum.

• DOI: 10.1111/jebm.12410
• 发表时间: 2020-08
• 期刊: Journal of evidence-based medicine

Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease.

• DOI: 10.1038/s41591-023-02636-6
• 发表时间: 2023-12
• 期刊: NATURE MEDICINE
• 影响因子: 82.9
• 作者: Chapman, Michael Spencer;Cull, Alyssa H.;Ciuculescu, Marioara F.;Esrick, Erica B.;Mitchell, Emily;Jung, Hyunchul;O'Neill, Laura;Roberts, Kirsty;Fabre, Margarete A.;Williams, Nicholas;Nangalia, Jyoti;Quinton, Joanne;Fox, James M.;Pellin, Danilo;Makani, Julie;Armant, Myriam;Williams, David A.;Campbell, Peter J.;Kent, David G.
• 通讯作者: Kent, David G.

Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling.

• DOI: 10.1038/s41467-023-40923-5
• 发表时间: 2023-09-20
• 期刊: NATURE COMMUNICATIONS
• 影响因子: 16.6
• 作者: De Souza, Daniel C.;Hebert, Nicolas;Esrick, Erica B.;Ciuculescu, M. Felicia;Archer, Natasha M.;Armant, Myriam;Audureau, Etienne;Brendel, Christian;Di Caprio, Giuseppe;Galacteros, Frederic;Liu, Donghui;Mccabe, Amanda;Morris, Emily;Schonbrun, Ethan;Williams, Dillon;Wood, David K.;Williams, David A.;Bartolucci, Pablo;Higgins, John M.
• 通讯作者: Higgins, John M.