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313 results

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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: schwartz jd. 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.
Leukocyte adhesion deficiency-I: A comprehensive review of all published cases.
Almarza Novoa E, Kasbekar S, Thrasher AJ, Kohn DB, Sevilla J, Nguyen T, Schwartz JD, Bueren JA. Almarza Novoa E, et al. Among authors: schwartz jd. J Allergy Clin Immunol Pract. 2018 Jul-Aug;6(4):1418-1420.e10. doi: 10.1016/j.jaip.2017.12.008. Epub 2018 Jan 20. J Allergy Clin Immunol Pract. 2018. PMID: 29371071 Free article. Review. No abstract available.
Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia.
Río P, Navarro S, Wang W, Sánchez-Domínguez R, Pujol RM, Segovia JC, Bogliolo M, Merino E, Wu N, Salgado R, Lamana ML, Yañez RM, Casado JA, Giménez Y, Román-Rodríguez FJ, Álvarez L, Alberquilla O, Raimbault A, Guenechea G, Lozano ML, Cerrato L, Hernando M, Gálvez E, Hladun R, Giralt I, Barquinero J, Galy A, García de Andoín N, López R, Catalá A, Schwartz JD, Surrallés J, Soulier J, Schmidt M, Díaz de Heredia C, Sevilla J, Bueren JA. Río P, et al. Among authors: schwartz jd. Nat Med. 2019 Sep;25(9):1396-1401. doi: 10.1038/s41591-019-0550-z. Epub 2019 Sep 9. Nat Med. 2019. PMID: 31501599 Clinical Trial.
Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal.
Moscatelli I, Almarza E, Schambach A, Ricks D, Schulz A, Herzog CD, Henriksen K, Askmyr M, Schwartz JD, Richter J. Moscatelli I, et al. Among authors: schwartz jd. Mol Ther Methods Clin Dev. 2020 Dec 25;20:389-397. doi: 10.1016/j.omtm.2020.12.009. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33575431 Free PMC article. Review.
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: schwartz jd. 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.
Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes.
Sevilla J, Navarro S, Rio P, Sánchez-Domínguez R, Zubicaray J, Gálvez E, Merino E, Sebastián E, Azqueta C, Casado JA, Segovia JC, Alberquilla O, Bogliolo M, Román-Rodríguez FJ, Giménez Y, Larcher L, Salgado R, Pujol RM, Hladun R, Castillo A, Soulier J, Querol S, Fernández J, Schwartz J, García de Andoín N, López R, Catalá A, Surralles J, Díaz-de-Heredia C, Bueren JA. Sevilla J, et al. Mol Ther Methods Clin Dev. 2021 Jun 12;22:66-75. doi: 10.1016/j.omtm.2021.06.001. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34485595 Free PMC 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. 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.
Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I.
Mesa-Núñez C, Damián C, Fernández-García M, Díez B, Rao G, Schwartz JD, Law KM, Sevilla J, Río P, Yáñez R, Bueren JA, Almarza E. Mesa-Núñez C, et al. Among authors: schwartz jd. Mol Ther Methods Clin Dev. 2022 Aug 1;26:459-470. doi: 10.1016/j.omtm.2022.07.015. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 36092365 Free PMC article.
313 results