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Page 1
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: bastone a. 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.
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: bastone al. 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.
Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning.
Schwarzer A, Talbot SR, Selich A, Morgan M, Schott JW, Dittrich-Breiholz O, Bastone AL, Weigel B, Ha TC, Dziadek V, Gijsbers R, Thrasher AJ, Staal FJT, Gaspar HB, Modlich U, Schambach A, Rothe M. Schwarzer A, et al. Among authors: bastone al. Mol Ther. 2021 Dec 1;29(12):3383-3397. doi: 10.1016/j.ymthe.2021.06.017. Epub 2021 Jun 24. Mol Ther. 2021. PMID: 34174440 Free PMC article.
TGFβ Inhibitor A83-01 Enhances Murine HSPC Expansion for Gene Therapy.
Fleischauer J, Bastone AL, Selich A, John-Neek P, Weisskoeppel L, Schaudien D, Schambach A, Rothe M. Fleischauer J, et al. Among authors: bastone al. Cells. 2023 Jul 31;12(15):1978. doi: 10.3390/cells12151978. Cells. 2023. PMID: 37566057 Free PMC article.
The usefulness of patient-reported outcome measures in decision making in Chagas cardiomyopathy: a scoping review.
Silva WT, Silva KLS, Xavier DM, Ribeiro Ávila M, de Oliveira LFF, Colicchio VM, de Almeida ILGI, Bastone AC, Alcantara MA, Lacerda ACR, Lima VP, de Oliveira LFL, Mediano MFF, Figueiredo PHS, Costa HS. Silva WT, et al. Among authors: bastone ac. Trans R Soc Trop Med Hyg. 2024 Aug 5;118(8):491-497. doi: 10.1093/trstmh/trae025. Trans R Soc Trop Med Hyg. 2024. PMID: 38717386 Review.
[What affects diagnostic and therapeutic times for cancer patients care?].
Coluccia S, Di Gennaro P, Crispo A, Grimaldi M, Nocerino F, Celentano E, Baglio G, Bastone A, Rivieccio G, Schiavone F, Bianchi AM, D'Errico D, Pignata S, Coppola E. Coluccia S, et al. Among authors: bastone a. Recenti Prog Med. 2023 Jun;114(6):362-367. doi: 10.1701/4042.40230. Recenti Prog Med. 2023. PMID: 37229684 Italian.
87 results