These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
7. Cellular function reinstitution of offspring red blood cells cloned from the sickle cell disease patient blood post CRISPR genome editing. Wen J; Tao W; Hao S; Zu Y J Hematol Oncol; 2017 Jun; 10(1):119. PubMed ID: 28610635 [TBL] [Abstract][Full Text] [Related]
8. Exagamglogene Autotemcel: First Approval. Hoy SM Mol Diagn Ther; 2024 Mar; 28(2):133-139. PubMed ID: 38228954 [TBL] [Abstract][Full Text] [Related]
9. Editorial: First Regulatory Approvals for CRISPR-Cas9 Therapeutic Gene Editing for Sickle Cell Disease and Transfusion-Dependent β-Thalassemia. Parums DV Med Sci Monit; 2024 Mar; 30():e944204. PubMed ID: 38425279 [TBL] [Abstract][Full Text] [Related]
10. Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States. Herring WL; Gallagher ME; Shah N; Morse KC; Mladsi D; Dong OM; Chawla A; Leiding JW; Zhang L; Paramore C; Andemariam B Pharmacoeconomics; 2024 Jun; 42(6):693-714. PubMed ID: 38684631 [TBL] [Abstract][Full Text] [Related]
14. Post-Transcriptional Genetic Silencing of Esrick EB; Lehmann LE; Biffi A; Achebe M; Brendel C; Ciuculescu MF; Daley H; MacKinnon B; Morris E; Federico A; Abriss D; Boardman K; Khelladi R; Shaw K; Negre H; Negre O; Nikiforow S; Ritz J; Pai SY; London WB; Dansereau C; Heeney MM; Armant M; Manis JP; Williams DA N Engl J Med; 2021 Jan; 384(3):205-215. PubMed ID: 33283990 [TBL] [Abstract][Full Text] [Related]
15. Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia. Ye L; Wang J; Tan Y; Beyer AI; Xie F; Muench MO; Kan YW Proc Natl Acad Sci U S A; 2016 Sep; 113(38):10661-5. PubMed ID: 27601644 [TBL] [Abstract][Full Text] [Related]
16. Specificity of CRISPR-Cas9 Editing in Exagamglogene Autotemcel. Yen A; Zappala Z; Fine RS; Majarian TD; Sripakdeevong P; Altshuler D N Engl J Med; 2024 May; 390(18):1723-1725. PubMed ID: 38657268 [No Abstract] [Full Text] [Related]
17. Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin. Ravi NS; Wienert B; Wyman SK; Bell HW; George A; Mahalingam G; Vu JT; Prasad K; Bandlamudi BP; Devaraju N; Rajendiran V; Syedbasha N; Pai AA; Nakamura Y; Kurita R; Narayanasamy M; Balasubramanian P; Thangavel S; Marepally S; Velayudhan SR; Srivastava A; DeWitt MA; Crossley M; Corn JE; Mohankumar KM Elife; 2022 Feb; 11():. PubMed ID: 35147495 [TBL] [Abstract][Full Text] [Related]
18. Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells. DeWitt MA; Magis W; Bray NL; Wang T; Berman JR; Urbinati F; Heo SJ; Mitros T; Muñoz DP; Boffelli D; Kohn DB; Walters MC; Carroll D; Martin DI; Corn JE Sci Transl Med; 2016 Oct; 8(360):360ra134. PubMed ID: 27733558 [TBL] [Abstract][Full Text] [Related]
19. Safety and efficacy of targeted busulfan therapy in children undergoing myeloablative matched sibling donor BMT for sickle cell disease. McPherson ME; Hutcherson D; Olson E; Haight AE; Horan J; Chiang KY Bone Marrow Transplant; 2011 Jan; 46(1):27-33. PubMed ID: 20305698 [TBL] [Abstract][Full Text] [Related]
20. Outcomes of matched sibling donor hematopoietic stem cell transplantation for severe sickle cell disease with myeloablative conditioning and intermediate-dose of rabbit anti-thymocyte globulin. Soni S; Gross TG; Rangarajan H; Baker KS; Sturm M; Rhodes M Pediatr Blood Cancer; 2014 Sep; 61(9):1685-9. PubMed ID: 24740582 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]