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Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.
Lessard S, Rimmelé P, Ling H, Moran K, Vieira B, Lin YD, Rajani GM, Hong V, Reik A, Boismenu R, Hsu B, Chen M, Cockroft BM, Uchida N, Tisdale J, Alavi A, Krishnamurti L, Abedi M, Galeon I, Reiner D, Wang L, Ramezi A, Rendo P, Walters MC, Levasseur D, Peters R, Harris T, Hicks A. Lessard S, et al. Sci Rep. 2024 Oct 16;14(1):24298. doi: 10.1038/s41598-024-74716-7. Sci Rep. 2024. PMID: 39414860 Free PMC article. Clinical Trial.
Metabolic blood profile and response to treatment with the pyruvate kinase activator mitapivat in patients with sickle cell disease.
van Dijk MJ, Ruiter TJJ, van der Veen S, Rab MAE, van Oirschot BA, Bos J, Derichs C, Rijneveld AW, Cnossen MH, Nur E, Biemond BJ, Bartels M, Schutgens REG, van Solinge WW, Jans JJM, van Beers EJ, van Wijk R. van Dijk MJ, et al. Hemasphere. 2024 Jun 25;8(6):e109. doi: 10.1002/hem3.109. eCollection 2024 Jun. Hemasphere. 2024. PMID: 38919958 Free PMC article.
Neurocognitive outcome in children with sickle cell disease after myeloimmunoablative conditioning and haploidentical hematopoietic stem cell transplantation: a non-randomized clinical trial.
Braniecki S, Vichinsky E, Walters MC, Shenoy S, Shi Q, Moore TB, Talano JA, Parsons SK, Flower A, Panarella A, Fabricatore S, Morris E, Mahanti H, Milner J, McKinstry RC, Duncan CN, van de Ven C, Cairo MS. Braniecki S, et al. Front Neurol. 2024 May 22;15:1263373. doi: 10.3389/fneur.2024.1263373. eCollection 2024. Front Neurol. 2024. PMID: 38841694 Free PMC article.
Genetics of Neonatal Lupus Erythematosus Risk and Specific Manifestations.
Misztal MC, Gold N, Cao J, Diaz T, Dominguez D, Thompson K, Jaeggi E, Knight AM, Laskin C, Ng L, Silverman ED, Hiraki LT. Misztal MC, et al. J Rheumatol. 2024 Jul 15:jrheum.2023-1036. doi: 10.3899/jrheum.2023-1036. Online ahead of print. J Rheumatol. 2024. PMID: 38825356
Exagamglogene Autotemcel for Severe Sickle Cell Disease.
Frangoul H, Locatelli F, Sharma A, Bhatia M, Mapara M, Molinari L, Wall D, Liem RI, Telfer P, Shah AJ, Cavazzana M, Corbacioglu S, Rondelli D, Meisel R, Dedeken L, Lobitz S, de Montalembert M, Steinberg MH, Walters MC, Eckrich MJ, Imren S, Bower L, Simard C, Zhou W, Xuan F, Morrow PK, Hobbs WE, Grupp SA; CLIMB SCD-121 Study Group. Frangoul H, et al. N Engl J Med. 2024 May 9;390(18):1649-1662. doi: 10.1056/NEJMoa2309676. Epub 2024 Apr 24. N Engl J Med. 2024. PMID: 38661449 Clinical Trial.
Machine Learning-Based Perivascular Space Volumetry in Alzheimer Disease.
Deike K, Decker A, Scheyhing P, Harten J, Zimmermann N, Paech D, Peters O, Freiesleben SD, Schneider LS, Preis L, Priller J, Spruth E, Altenstein S, Lohse A, Fliessbach K, Kimmich O, Wiltfang J, Bartels C, Hansen N, Jessen F, Rostamzadeh A, Düzel E, Glanz W, Incesoy EI, Butryn M, Buerger K, Janowitz D, Ewers M, Perneczky R, Rauchmann BS, Teipel S, Kilimann I, Goerss D, Laske C, Munk MH, Spottke A, Roy N, Wagner M, Roeske S, Heneka MT, Brosseron F, Ramirez A, Dobisch L, Wolfsgruber S, Kleineidam L, Yakupov R, Stark M, Schmid MC, Berger M, Hetzer S, Dechent P, Scheffler K, Petzold GC, Schneider A, Effland A, Radbruch A. Deike K, et al. Invest Radiol. 2024 Sep 1;59(9):667-676. doi: 10.1097/RLI.0000000000001077. Epub 2024 Apr 23. Invest Radiol. 2024. PMID: 38652067
Transcription-coupled DNA-protein crosslink repair by CSB and CRL4CSA-mediated degradation.
van Sluis M, Yu Q, van der Woude M, Gonzalo-Hansen C, Dealy SC, Janssens RC, Somsen HB, Ramadhin AR, Dekkers DHW, Wienecke HL, Demmers JJPG, Raams A, Davó-Martínez C, Llerena Schiffmacher DA, van Toorn M, Häckes D, Thijssen KL, Zhou D, Lammers JG, Pines A, Vermeulen W, Pothof J, Demmers JAA, van den Berg DLC, Lans H, Marteijn JA. van Sluis M, et al. Nat Cell Biol. 2024 May;26(5):770-783. doi: 10.1038/s41556-024-01394-y. Epub 2024 Apr 10. Nat Cell Biol. 2024. PMID: 38600236 Free PMC article.
149 results