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141 results

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Page 1
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
Castiello MC, Brandas C, Ferrari S, Porcellini S, Sacchetti N, Canarutto D, Draghici E, Merelli I, Barcella M, Pelosi G, Vavassori V, Varesi A, Jacob A, Scala S, Basso Ricci L, Paulis M, Strina D, Di Verniere M, Sergi Sergi L, Serafini M, Holland SM, Bergerson JRE, De Ravin SS, Malech HL, Pala F, Bosticardo M, Brombin C, Cugnata F, Calzoni E, Crooks GM, Notarangelo LD, Genovese P, Naldini L, Villa A. Castiello MC, et al. Among authors: crooks gm. Sci Transl Med. 2024 Feb 7;16(733):eadh8162. doi: 10.1126/scitranslmed.adh8162. Epub 2024 Feb 7. Sci Transl Med. 2024. PMID: 38324638 Free PMC article.
Perivascular support of human hematopoietic stem/progenitor cells.
Corselli M, Chin CJ, Parekh C, Sahaghian A, Wang W, Ge S, Evseenko D, Wang X, Montelatici E, Lazzari L, Crooks GM, Péault B. Corselli M, et al. Among authors: crooks gm. Blood. 2013 Apr 11;121(15):2891-901. doi: 10.1182/blood-2012-08-451864. Epub 2013 Feb 14. Blood. 2013. PMID: 23412095 Free PMC article.
From pluripotent stem cells to T cells.
Montel-Hagen A, Crooks GM. Montel-Hagen A, et al. Among authors: crooks gm. Exp Hematol. 2019 Mar;71:24-31. doi: 10.1016/j.exphem.2018.12.001. Epub 2018 Dec 24. Exp Hematol. 2019. PMID: 30590093 Free article. Review.
Artificial thymic organoids represent a reliable tool to study T-cell differentiation in patients with severe T-cell lymphopenia.
Bosticardo M, Pala F, Calzoni E, Delmonte OM, Dobbs K, Gardner CL, Sacchetti N, Kawai T, Garabedian EK, Draper D, Bergerson JRE, DeRavin SS, Freeman AF, Güngör T, Hartog N, Holland SM, Kohn DB, Malech HL, Markert ML, Weinacht KG, Villa A, Seet CS, Montel-Hagen A, Crooks GM, Notarangelo LD. Bosticardo M, et al. Among authors: crooks gm. Blood Adv. 2020 Jun 23;4(12):2611-2616. doi: 10.1182/bloodadvances.2020001730. Blood Adv. 2020. PMID: 32556283 Free PMC article.
Gene Editing Rescues In vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System.
Gardner CL, Pavel-Dinu M, Dobbs K, Bosticardo M, Reardon PK, Lack J, DeRavin SS, Le K, Bello E, Pala F, Delmonte OM, Malech H, Montel-Hagan A, Crooks G, Acuto O, Porteus MH, Notarangelo LD. Gardner CL, et al. J Clin Immunol. 2021 Jul;41(5):852-862. doi: 10.1007/s10875-021-00989-6. Epub 2021 Mar 1. J Clin Immunol. 2021. PMID: 33650026 Free PMC article.
Correction to: Gene Editing Rescues in Vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System.
Gardner CL, Pavel-Dinu M, Dobbs K, Bosticardo M, Reardon PK, Lack J, DeRavin SS, Le K, Bello E, Pala F, Delmonte OM, Malech H, Montel-Hagen A, Crooks G, Acuto O, Porteus MH, Notarangelo LD. Gardner CL, et al. J Clin Immunol. 2021 Jul;41(5):863. doi: 10.1007/s10875-021-01030-6. J Clin Immunol. 2021. PMID: 33821398 No abstract available.
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. Kohn DB, et al. Among authors: crooks gm. N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11. N Engl J Med. 2021. PMID: 33974366 Free PMC article. Clinical Trial.
T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.
Kohn DB, Hershfield MS, Carbonaro D, Shigeoka A, Brooks J, Smogorzewska EM, Barsky LW, Chan R, Burotto F, Annett G, Nolta JA, Crooks G, Kapoor N, Elder M, Wara D, Bowen T, Madsen E, Snyder FF, Bastian J, Muul L, Blaese RM, Weinberg K, Parkman R. Kohn DB, et al. Nat Med. 1998 Jul;4(7):775-80. doi: 10.1038/nm0798-775. Nat Med. 1998. PMID: 9662367 Free PMC article.
141 results