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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: benedetti s. 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.
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: benedetti s. 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.
313 results