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Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA.
Ellison SM, Liao A, Wood S, Taylor J, Youshani AS, Rowlston S, Parker H, Armant M, Biffi A, Chan L, Farzaneh F, Wynn R, Jones SA, Heal P, Gaspar HB, Bigger BW. Ellison SM, et al. Among authors: gaspar hb. Mol Ther Methods Clin Dev. 2019 Apr 6;13:399-413. doi: 10.1016/j.omtm.2019.04.001. eCollection 2019 Jun 14. Mol Ther Methods Clin Dev. 2019. PMID: 31044143 Free PMC article.
The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity.
Montiel-Equihua CA, Zhang L, Knight S, Saadeh H, Scholz S, Carmo M, Alonso-Ferrero ME, Blundell MP, Monkeviciute A, Schulz R, Collins M, Takeuchi Y, Schmidt M, Fairbanks L, Antoniou M, Thrasher AJ, Gaspar HB. Montiel-Equihua CA, et al. Among authors: gaspar hb. Mol Ther. 2012 Jul;20(7):1400-9. doi: 10.1038/mt.2012.50. Epub 2012 Mar 20. Mol Ther. 2012. PMID: 22434141 Free PMC article.
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Eichler F, et al. Among authors: gaspar hb. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4. N Engl J Med. 2017. PMID: 28976817 Free PMC article. Clinical Trial.
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: gaspar hb. 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.
Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.
Garcia-Perez L, van Eggermond M, van Roon L, Vloemans SA, Cordes M, Schambach A, Rothe M, Berghuis D, Lagresle-Peyrou C, Cavazzana M, Zhang F, Thrasher AJ, Salvatori D, Meij P, Villa A, Van Dongen JJM, Zwaginga JJ, van der Burg M, Gaspar HB, Lankester A, Staal FJT, Pike-Overzet K. Garcia-Perez L, et al. Among authors: gaspar hb. Mol Ther Methods Clin Dev. 2020 Mar 31;17:666-682. doi: 10.1016/j.omtm.2020.03.016. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32322605 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: gaspar hb. 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.
Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.
Yeung J, Liao A, Shaw M, Silva S, Vetharoy W, Rico DL, Kirby I, Zammarchi F, Havenith K, de Haan L, van Berkel PH, Sebire N, Ogunbiyi OK, Booth C, Gaspar HB, Thrasher AJ, Chester KA, Amrolia PJ. Yeung J, et al. Among authors: gaspar hb. Mol Ther. 2024 Jun 5;32(6):1672-1686. doi: 10.1016/j.ymthe.2024.03.032. Epub 2024 Mar 27. Mol Ther. 2024. PMID: 38549377
197 results