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Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment.
Navarro S, Quintana-Bustamante O, Sanchez-Dominguez R, Lopez-Manzaneda S, Ojeda-Perez I, Garcia-Torralba A, Alberquilla O, Law K, Beard BC, Bastone A, Rothe M, Villanueva M, Ramirez JC, Fañanas-Baquero S, Nieto-Romero V, Molinos-Vicente A, Gutierrez S, Nicoletti E, García-Bravo M, Bueren JA, Schwartz JD, Segovia JC. Navarro S, et al. Among authors: nicoletti e. Mol Ther Methods Clin Dev. 2021 Jul 29;22:350-359. doi: 10.1016/j.omtm.2021.07.006. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34514027 Free PMC article.
Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients.
Ramírez MJ, Pujol R, Trujillo-Quintero JP, Minguillón J, Bogliolo M, Río P, Navarro S, Casado JA, Badell I, Carrasco E, Balmaña J, Català A, Sevilla J, Beléndez C, Argilés B, López M, Díaz de Heredia C, Rao G, Nicoletti E, Schwartz JD, Bueren JA, Surrallés J. Ramírez MJ, et al. Among authors: nicoletti e. Am J Hematol. 2021 Aug 1;96(8):989-999. doi: 10.1002/ajh.26234. Epub 2021 May 25. Am J Hematol. 2021. PMID: 33984160 Free article.
Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD.
Schwartz JD, Barcellini W, Grace RF, Bianchi P, Zanella A, López Lorenzo JL, Sevilla J, Shah AJ, Glader B, Nicoletti E, Navarro Ordoñez S, Segovia JC. Schwartz JD, et al. Among authors: nicoletti e. Am J Hematol. 2022 Mar 1;97(3):E120-E125. doi: 10.1002/ajh.26458. Epub 2022 Jan 24. Am J Hematol. 2022. PMID: 34989415 Free PMC article. Review. No abstract available.
Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia.
Lasaga M, Río P, Vilas-Zornoza A, Planell N, Navarro S, Alignani D, Fernández-Varas B, Mouzo D, Zubicaray J, Pujol RM, Nicoletti E, Schwartz JD, Sevilla J, Ainciburi M, Ullate-Agote A, Surrallés J, Perona R, Sastre L, Prosper F, Gomez-Cabrero D, Bueren JA. Lasaga M, et al. Among authors: nicoletti e. Haematologica. 2023 Oct 1;108(10):2652-2663. doi: 10.3324/haematol.2022.282418. Haematologica. 2023. PMID: 37021532 Free PMC article.
Busulfan and subsequent malignancy: An evidence-based risk assessment.
Long-Boyle JR, Kohn DB, Shah AJ, Spencer SM, Sevilla J, Booth C, López Lorenzo JL, Nicoletti E, Shah A, Reatz M, Matos J, Schwartz JD. Long-Boyle JR, et al. Among authors: nicoletti e. Pediatr Blood Cancer. 2024 Jan;71(1):e30738. doi: 10.1002/pbc.30738. Epub 2023 Oct 19. Pediatr Blood Cancer. 2024. PMID: 37856098
Findings on Optical Coherence Tomography in Malignant Infantile Osteopetrosis.
Campagna GA, Chew L, Pettenkofer M, Nicoletti E, Schwartz JD, Choi G, Fernandes AO, Terrazas D, Kohn DB, Tsui I. Campagna GA, et al. Among authors: nicoletti e. Ophthalmic Surg Lasers Imaging Retina. 2022 Jul;53(7):398-402. doi: 10.3928/23258160-20220613-01. Epub 2022 Jul 1. Ophthalmic Surg Lasers Imaging Retina. 2022. PMID: 35858232
Donor-Host Lineage-Specific Chimerism Monitoring and Analysis in Pediatric Patients Following Allogeneic Stem Cell Transplantation: Influence of Pretransplantation Variables and Correlation with Post-Transplantation Outcomes.
Llaurador G, Nicoletti E, Prockop SE, Hsu S, Fuller K, Mauguen A, O'Reilly RJ, Boelens JJ, Boulad F. Llaurador G, et al. Among authors: nicoletti e. Transplant Cell Ther. 2021 Sep;27(9):780.e1-780.e14. doi: 10.1016/j.jtct.2021.05.020. Epub 2021 May 31. Transplant Cell Ther. 2021. PMID: 34082161 Free PMC article.
Cross-cultural adaptation of the Internet Gaming Disorder Scale - Short Form (IGDS9-SF) to the Brazilian context.
Donadon MF, Chagas MHN, Apolinário-da-Silva TD, Okino ETK, Hallak JEC, Nicoletti ÊA, Pereira-Lima K, Degan EA, Santos RG, Machado-de-Sousa JP, Simei JLQ, Oliveira LM, Pontes HM, Osório FL. Donadon MF, et al. Among authors: nicoletti ea. Trends Psychiatry Psychother. 2020 Jul-Sep;42(3):262-266. doi: 10.1590/2237-6089-2019-0032. Trends Psychiatry Psychother. 2020. PMID: 32844978 Free PMC article.
24 results