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Systemic AAV9.LAMP2B injection reverses metabolic and physiologic multiorgan dysfunction in a murine model of Danon disease.
Manso AM, Hashem SI, Nelson BC, Gault E, Soto-Hermida A, Villarruel E, Brambatti M, Bogomolovas J, Bushway PJ, Chen C, Battiprolu P, Keravala A, Schwartz JD, Shah G, Gu Y, Dalton ND, Hammond K, Peterson K, Saftig P, Adler ED. Manso AM, et al. Among authors: schwartz jd. Sci Transl Med. 2020 Mar 18;12(535):eaax1744. doi: 10.1126/scitranslmed.aax1744. Sci Transl Med. 2020. PMID: 32188720
AAV-Mediated Delivery of Plakophilin-2a Arrests Progression of Arrhythmogenic Right Ventricular Cardiomyopathy in Murine Hearts: Preclinical Evidence Supporting Gene Therapy in Humans.
van Opbergen CJM, Narayanan B, Sacramento CB, Stiles KM, Mishra V, Frenk E, Ricks D, Chen G, Zhang M, Yarabe P, Schwartz J, Delmar M, Herzog CD, Cerrone M. van Opbergen CJM, et al. Circ Genom Precis Med. 2024 Feb;17(1):e004305. doi: 10.1161/CIRCGEN.123.004305. Epub 2024 Jan 30. Circ Genom Precis Med. 2024. PMID: 38288614 Free article.
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.
Mitapivat versus Placebo for Pyruvate Kinase Deficiency.
Shah AJ, Schwartz JD, Segovia JC. Shah AJ, et al. Among authors: schwartz jd. N Engl J Med. 2022 Jun 30;386(26):2538-2539. doi: 10.1056/NEJMc2206275. N Engl J Med. 2022. PMID: 35767453 No abstract available.
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.
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.
314 results