Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease

Authors: Piras, Giuseppa, Montiel-Equihua, Claudia, Chan, Yee-Ka Agnes, Wantuch, Slawomir, Stuckey, Daniel, Burke, Derek, Prunty, Helen, Phadke, Rahul, Chambers, Darren, Partida-Gaytan, Armando, Leon-Rico, Diego, Panchal, Neelam, Whitmore, Kathryn, Calero, Miguel, Benedetti, Sara, Santilli, Giorgia, Thrasher, Adrian J., Gaspar, H. Bobby

Source: In Molecular Therapy - Methods & Clinical Development 11 September 2020 18:558-570

Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application

Authors: Schott, Juliane W., León-Rico, Diego, Ferreira, Carolina B., Buckland, Karen F., Santilli, Giorgia, Armant, Myriam A., Schambach, Axel, Cavazza, Alessia, Thrasher, Adrian J.

Source: In Molecular Therapy - Methods & Clinical Development 13 September 2019 14:134-147

The downregulated membrane expression of CD18 in CD34+ cells defines a primitive population of human hematopoietic stem cells.

Authors: Mesa-Núñez, Cristina^1,2,3 (AUTHOR), Leon-Rico, Diego^1,2,3 (AUTHOR), Aldea, Montserrat^1,2,3 (AUTHOR), Damián, Carlos^1,2,3 (AUTHOR), Sanchez-Baltasar, Raquel^1,2,3 (AUTHOR), Sanchez, Rebeca^1,2,3 (AUTHOR), Alberquilla, Omaira^1,2,3 (AUTHOR), Segovia, José Carlos^1,2,3 (AUTHOR), Bueren, Juan Antonio^1,2,3 (AUTHOR), Almarza, Elena^1,2,3 (AUTHOR) elenaalmarza@gmail.com

Source: Stem Cell Research & Therapy. 4/28/2020, Vol. 11 Issue 1, p1-9. 9p.

Subject Terms: *HUMAN stem cells, *HEMATOPOIETIC stem cells, *CORD blood, *CELL adhesion, *CELLS, *BONE marrow, *INTEGRINS

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Authors: Booth, Claire ^*, Kohn, Donald B., Armant, Myriam ^*, Prockop, Susan, Buckland, Karen ^*, Chandra, Sharat ^*, Chandrakasan, Shanmuganathan, Chetty, Kritika ^*, Dansereau, Colleen ^*, de Oliveira, Satiro ^*, Everett, John ^*, León Rico, Diego ^*, London, Wendy B., Xu-Bayford, Jin Hua ^*, Moore, Theodore B., Nagarsheth, Nisha ^*, Parikh, Suhag ^*, Terrazas, Dayna ^*, White, Shanna L ^*, Schambach, Axel ^*, Bushman, Frederic D. ^*, Thrasher, Adrian J. ^*, Williams, David A., Pai, Sung-Yun

Source: In Blood 15 November 2022 140 Supplement 1:7770-7771

Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47^phox-Deficient Chronic Granulomatous Disease.

Authors: Schejtman, Andrea, Vetharoy, Winston, Choi, Uimook, Rivat, Christine, Theobald, Narda, Piras, Giuseppa, Leon-Rico, Diego, Buckland, Karen, Armenteros-Monterroso, Elena, Benedetti, Sara, Ashworth, Michael T., Rothe, Michael, Schambach, Axel, Gaspar, Hubert Bobby, Kang, Elizabeth M., Malech, Harry L., Thrasher, Adrian J., Santilli, Giorgia

Source: Human Gene Therapy; Sep2021, Vol. 32 Issue 17/18, p949-958, 10p

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome.

Authors: Rai, Rajeev, Romito, Marianna, Rivers, Elizabeth, Turchiano, Giandomenico, Blattner, Georges, Vetharoy, Winston, Ladon, Dariusz, Andrieux, Geoffroy, Zhang, Fang, Zinicola, Marta, Leon-Rico, Diego, Santilli, Giorgia, Thrasher, Adrian J., Cavazza, Alessia

Source: Nature Communications; 8/12/2020, Vol. 11 Issue 1, p1-15, 15p

Subject Terms: HEMATOPOIETIC stem cells, HUMAN stem cells, WISKOTT-Aldrich syndrome, STEM cell treatment, STEM cells, GENOME editing

Case report of the first patient treated with ex-vivo autologous haematopoietic stem cell gene therapy transplant in mucopolysaccharidosis type IIIA

Authors: Kinsella, Jane Louise, Church, Heather, Ogden, Wendy, Jones, Ceri, Buckland, Karen, Rivat, Christine, Thrasher, Adrian, Snell, Katie, Leon-Rico, Diego, Vaz, Fred, Wijburg, Frits, Jones, Simon, Bigger, Brian, Wynn, Robert

Source: In Molecular Genetics and Metabolism February 2020 129(2):S90-S90

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Lentiviral gene therapy for X-linked chronic granulomatous disease

Authors: Kohn, Donald B., Booth, Claire, Kang, Elizabeth M., Pai, Sung-Yun, Shaw, Kit L., Santilli, Giorgia, Armant, Myriam, Buckland, Karen F., Choi, Uimook, De Ravin, Suk See, Dorsey, Morna J., Kuo, Caroline Y., Leon-Rico, Diego, Rivat, Christine, Izotova, Natalia, Gilmour, Kimberly, Snell, Katie, Dip, Jinhua Xu-Bayford, Darwish, Jinan, Morris, Emma C., Terrazas, Dayna, Wang, Leo D., Bauser, Christopher A., Paprotka, Tobias, Kuhns, Douglas B., Gregg, John, Raymond, Hayley E., Everett, John K., Honnet, Geraldine, Biasco, Luca, Newburger, Peter E., Bushman, Frederic D., Grez, Manuel, Gaspar, H. Bobby, Williams, David A., Malech, Harry L., Galy, Anne, Thrasher, Adrian J.

Source: Nature Medicine; February 2020, Vol. 26 Issue: 2 p200-206, 7p

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