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Lentiviral gene therapy for X-linked chronic granulomatous disease.
Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ; Net4CGD consortium. Kohn DB, et al. Among authors: leon rico d. Nat Med. 2020 Feb;26(2):200-206. doi: 10.1038/s41591-019-0735-5. Epub 2020 Jan 27. Nat Med. 2020. PMID: 31988463 Free PMC article. Clinical Trial.
Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease.
Schejtman A, Vetharoy W, Choi U, Rivat C, Theobald N, Piras G, Leon-Rico D, Buckland K, Armenteros-Monterroso E, Benedetti S, Ashworth MT, Rothe M, Schambach A, Gaspar HB, Kang EM, Malech HL, Thrasher AJ, Santilli G. Schejtman A, et al. Among authors: leon rico d. Hum Gene Ther. 2021 Sep;32(17-18):949-958. doi: 10.1089/hum.2020.276. Epub 2021 May 6. Hum Gene Ther. 2021. PMID: 33740872 Free PMC article.
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. Kohn DB, et al. Among authors: leon rico d. N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11. N Engl J Med. 2021. PMID: 33974366 Free PMC article. Clinical Trial.
Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease.
Piras G, Montiel-Equihua C, Chan YA, Wantuch S, Stuckey D, Burke D, Prunty H, Phadke R, Chambers D, Partida-Gaytan A, Leon-Rico D, Panchal N, Whitmore K, Calero M, Benedetti S, Santilli G, Thrasher AJ, Gaspar HB. Piras G, et al. Among authors: leon rico d. Mol Ther Methods Clin Dev. 2020 Jul 6;18:558-570. doi: 10.1016/j.omtm.2020.07.001. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32775491 Free PMC article.
BCR-JAK2 drives a myeloproliferative neoplasm in transplanted mice.
Cuesta-Domínguez Á, León-Rico D, Álvarez L, Díez B, Bodega-Mayor I, Baños R, Martín-Rey MÁ, Santos-Roncero M, Gaspar ML, Martín-Acosta P, Almarza E, Bueren JA, Río P, Fernández-Ruiz E. Cuesta-Domínguez Á, et al. Among authors: leon rico d. J Pathol. 2015 Jun;236(2):219-28. doi: 10.1002/path.4513. Epub 2015 Mar 3. J Pathol. 2015. PMID: 25664618
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