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Year Number of Results
1996 3
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2008 1
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2025 4

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29 results

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Page 1
Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.
Booth C, Sevilla J, Almarza E, Kuo CY, Zubicaray J, Terrazas D, O'Toole G, Chitty-Lopez M, Choi G, Nicoletti E, Long-Boyle J, Fernandes A, Chetty K, De Oliveira S, Banuelos C, Xu-Bayford J, Bastone AL, John-Neek P, Jackson C, Moore TB, Gilmour K, Schambach A, Rothe M, Kasbekar S, Rao GR, Patel K, Shah G, Thrasher AJ, Bueren JA, Schwartz JD, Kohn DB. Booth C, et al. Among authors: nicoletti e. N Engl J Med. 2025 May 1;392(17):1698-1709. doi: 10.1056/NEJMoa2407376. N Engl J Med. 2025. PMID: 40305711 Clinical Trial.
Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
Río P, Zubicaray J, Navarro S, Gálvez E, Sánchez-Domínguez R, Nicoletti E, Sebastián E, Rothe M, Pujol R, Bogliolo M, John-Neek P, Bastone AL, Schambach A, Wang W, Schmidt M, Larcher L, Segovia JC, Yáñez RM, Alberquilla O, Díez B, Fernández-García M, García-García L, Ramírez M, Galy A, Lefrere F, Cavazzana M, Leblanc T, García de Andoin N, López-Almaraz R, Catalá A, Barquinero J, Rodríguez-Perales S, Rao G, Surrallés J, Soulier J, Díaz-de-Heredia C, Schwartz JD, Sevilla J, Bueren JA; FANCOLEN-1 gene therapy investigators. Río P, et al. Among authors: nicoletti e. Lancet. 2025 Dec 21;404(10471):2584-2592. doi: 10.1016/S0140-6736(24)01880-4. Epub 2024 Dec 3. Lancet. 2025. PMID: 39642902 Clinical Trial.
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.
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
Eltrombopag for Bone Marrow Failure in Fanconi Anemia: Results From the Phase II Clinical Trial FANCREV.
Iriondo J, Zubicaray J, Río P, Català A, Solsona M, Sanz A, Gómez A, Sebastián E, de la Cruz A, Galán A, Navarro S, Larcher L, de Andoin NG, Uriz JJ, Vagace JM, Gonzalez de Pablo J, Pujol MR, Nicoletti E, Surrallés J, Martín-Prado S, Schwartz JD, Soulier J, Bueren JA, Sevilla J. Iriondo J, et al. Among authors: nicoletti e. Eur J Haematol. 2025 Oct;115(4):403-412. doi: 10.1111/ejh.70007. Epub 2025 Jul 16. Eur J Haematol. 2025. PMID: 40665878 Clinical Trial.
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
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.
29 results