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Journal Articles

International Clinical Consensus on Leukocyte Adhesion Deficiency-I: A Modified Delphi Analysis

Jennifer Heimall, Luis Ignacio González Granado, Claire Booth, Patrice Carter, Emily Matthews, Maria Chitty-Lopez, Miranda Bailey, Susan E Prockop, Andrew R Gennery, Jennifer W Leiding
Journal: Blood
Blood (2024) 144 (Supplement 1): 3639.
https://doi.org/10.1182/blood-2024-207939
Published: 2024
Journal Articles

Ex Vivo Lentiviral Hematopoietic Stem Cell (HSC) Gene Therapy May Represent a Curative Therapy for the Life-Threatening Inborn Error of Immunity Severe Combined Immunodeficiency Due to Adenosine Deaminase (ADA) Deficiency (ADA-SCID)

Katelyn Masiuk, Jinhua Xu-Bayford, Augustine Fernandes, Konstantinos Vazouras, Danilo Pellin, Havinder Hara, Sohini Roy, Beatriz Campo Fernandez, Thao Thai Dang, Kimberly Gilmour, Elena Alvarez Mediavilla, Beatrice Curio-Penny, Gráinne O'Toole, Rima Ahmed, Jordyn Arnold, Elizabeth Garabedian, Harry L Malech, Kenneth Cornetta, Claire Booth, Donald B. Kohn
Journal: Blood
Blood (2024) 144 (Supplement 1): 1051.
https://doi.org/10.1182/blood-2024-202543
Published: 2024
Journal Articles

Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group a]: Updated Results from Global RP-L102 Clinical Trials

Agnieszka D. Czechowicz, Julián Sevilla, Claire Booth, Josune Zubicaray, Paula Rio, Susana Navarro, Kritika Cherry, Gráinne O'Toole, Jinhua Xu-Bayford, Philip Ancliff, Elena Sebastián, Michael Rothe, Antonella L Bastone, Philipp John-Neek, Axel Schambach, Wei Wang, Grace Choi, Miriam Zeini, Eileen Nicoletti, Cindy Eide, John E Wagner, Gayatri R Rao, Adrian J. Thrasher, Maria-Grazia Roncarolo, Jonathan D Schwartz, Juan A Bueren, Rajni Agarwal
Journal: Blood
Blood (2024) 144 (Supplement 1): 7463.
https://doi.org/10.1182/blood-2024-194638
Published: 2024
Journal Articles

Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) Outcomes in Leukocyte Adhesion Deficiency Type -I

Claire Booth, Patrick Gilbert, Luuk Gras, Hawazen Alsaedi, Amir Ali Hamidieh, Mor Seny Gueye, Mary Slatter, Ashrafsadat Mousavi, Amos Toren, Musa Karakukcu, Abdelghani Tbakhi, Polina Stepensky, Gulyuz Ozturk, Peter Bader, Henrik Sengeloev, Tayfun Güngör, R.F. Wynn, Franco Locatelli, Maria Chitty-Lopez, Joana E Matos, Stuart Turner, Gayatri R Rao, Jonathan D Schwartz, Michael H Albert, Bénédicte Neven
Journal: Blood
Blood (2024) 144 (Supplement 1): 5079.
https://doi.org/10.1182/blood-2024-203643
Published: 2024
Journal Articles

Biochemical Engraftment and Clinical Outcomes Following Ex-Vivo Autologous Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA

Robert F Wynn, Jane L Kinsella, Rebecca J Holley, Jane Potter, Adrian J. Thrasher, Claire Booth, Karen Buckland, Natalia Izotova, Stewart Rust, Daniel Weisberg, Heather J Church, Karen L Tylee, Kathryn L Brammeier, Helena Lee, Laura Ford, Atusa Sadegholnejat, Simon A Jones, Stuart Ellison, Brian Bigger
Journal: Blood
Blood (2022) 140 (Supplement 1): 1897–1898.
https://doi.org/10.1182/blood-2022-168672
Published: 2022
Journal Articles

Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials

Agnieszka Czechowicz, Julián Sevilla, Claire Booth, Rajni Agarwal, Josune Zubicaray, Paula Río, Susana Navarro, Kritika Chetty, Grainne O'Toole, Jinhua Xu-Bayford, Philip Ancliff, Elena Sebastián, Grace Choi, Miriam Zeini, Eileen Nicoletti, John E Wagner, Gayatri R. Rao, Adrian J. Thrasher, Jonathan D. Schwartz, Maria Grazia Roncarolo, Juan A. Bueren
Journal: Blood
Blood (2022) 140 (Supplement 1): 10646–10647.
https://doi.org/10.1182/blood-2022-168342
Published: 2022
Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Sung-Yun Pai, Nisha Nagarsheth, Susan Prockop, Myriam Armant, Donald B. Kohn, Rebecca A. Marsh, Claire Booth, John K. Everett, Jack Bleesing, Deepak Chellapandian, Colleen Dansereau, Satiro de Oliveira, Wendy B. London, M. Angélica Marinovic, Theodore B. Moore, Matias Oleastro, Grainne O'Toole, Mark Vander Lugt, Luciano Urdinez, Kelly J. Walkovich, Jolan E. Walter, Axel Schambach, Adrian J. Thrasher, Frederic D. Bushman, David A Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10643–10645.
https://doi.org/10.1182/blood-2022-159768
Published: 2022
Journal Articles

Development of Anti-CD45 Antibody Drug Conjugates As Targeted Conditioning Agents for Transplantation/Gene Therapy with Potent Anti-Leukemic Properties

Jenny Yeung, Aiyin Liao, Matthew Shaw, Soraia Caetano-Silva, Winston Vetharoy, Ian Kirby, Francesca Zammarchi, Karin Havenith, Patrick H van Berkel, Claire Booth, H Bobby Gaspar, Adrian J. Thrasher, Kerry Chester, Persis J Amrolia
Journal: Blood
Blood (2022) 140 (Supplement 1): 10211–10212.
https://doi.org/10.1182/blood-2022-162390
Published: 2022
Journal Articles

Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Claire Booth, Julián Sevilla, Gayatri R. Rao, Maria Chitty Lopez, Elena Almarza, Dayna Terrazas, Josune Zubicaray, Marta González Vicent, Kritika Chetty, Grainne O'Toole, Jinhua Xu-Bayford, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A. Bueren, Jonathan D. Schwartz, Donald B. Kohn
Journal: Blood
Blood (2022) 140 (Supplement 1): 7774–7775.
https://doi.org/10.1182/blood-2022-158578
Published: 2022
Journal Articles

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

Claire Booth, Donald B. Kohn, Myriam Armant, Susan Prockop, Karen Buckland, Sharat Chandra, Shanmuganathan Chandrakasan, Kritika Chetty, Colleen Dansereau, Satiro de Oliveira, John Everett, Diego León Rico, Wendy B. London, Jin Hua Xu-Bayford, Theodore B. Moore, Nisha Nagarsheth, Suhag Parikh, Dayna Terrazas, Shanna L White, Axel Schambach, Frederic D. Bushman, Adrian J. Thrasher, David A. Williams, Sung-Yun Pai
Journal: Blood
Blood (2022) 140 (Supplement 1): 7770–7771.
https://doi.org/10.1182/blood-2022-159979
Published: 2022
Journal Articles

Quercetin ameliorates XIAP deficiency–associated hyperinflammation

Samuel C. C. Chiang, Erika Owsley, Neelam Panchal, Vijaya Chaturvedi, Catherine E. Terrell, Michael B. Jordan, Parinda A. Mehta, Stella M. Davies, Nagako Akeno, Claire Booth, Rebecca A. Marsh
Journal: Blood
Blood (2022) 140 (7): 706–715.
https://doi.org/10.1182/blood.2021014335
Published: 2022
Includes: Supplemental data
Journal Articles

Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials

Agnieszka Czechowicz, Julián Sevilla, Rajni Agarwal, Claire Booth, Josune Zubicaray, Paula Río, Susana Navarro, Philip Ancliff, Elena Sebastian, Brian C Beard, Kenneth M Law, Grace Choi, Miriam Zeini, Camilla Duran-Persson, Eileen Nicoletti, John E Wagner, Gayatri R Rao, Adrian J. Thrasher, Jonathan D Schwartz, Juan A Bueren, Maria Grazia Roncarolo
Journal: Blood
Blood (2021) 138 (Supplement 1): 3968.
https://doi.org/10.1182/blood-2021-147071
Published: 2021
Journal Articles

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Donald B. Kohn, Claire Booth, Julián Sevilla, Gayatri R Rao, Elena Almarza, Dayna Terrazas, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Kenneth M Law, Brian C Beard, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A Bueren, Jonathan D Schwartz
Journal: Blood
Blood (2021) 138 (Supplement 1): 2932.
https://doi.org/10.1182/blood-2021-151898
Published: 2021
Journal Articles

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Alessandra Magnani, Michaela Semeraro, Frédéric ADAM, Claire Booth, Loic Dupre, Emma C Morris, Aurélie Gabrion, Cecile Roudaut, Delphine Borgel, Antoine Toubert, Emmanuel Clave, Chrystelle Abdo, Guy Gorochov, Rachel Petermann, Mélanie Guiot, Makoto Miyara, Despina Moshous, Elisa Magrin, Adeline Denis, Felipe Suarez, Chantal Lagresle-Peyrou, Aoife Doto, John K. Everett, Amélie Trinquand, Marianne Guisset, Jin Hua Xu-Bayford, Salima Hacein-Bey-Abina, Alexandre Kauskot, Reem Elfeky, Christine Rivat, Sarah Abbas, Bobby H Gaspar, Elizabeth A. Macintyre, Capucine Picard, Frederic D. Bushman, Anne Galy, Alain Fischer, Emmanuelle Six, Adrian J. Thrasher, Marina Cavazzana
Journal: Blood
Blood (2021) 138 (Supplement 1): 2934.
https://doi.org/10.1182/blood-2021-147697
Published: 2021
Journal Articles

Absent B cells, agammaglobulinemia, and hypertrophic cardiomyopathy in folliculin-interacting protein 1 deficiency

Brief Report
Francesco Saettini, Cecilia Poli, Jaime Vengoechea, Sonia Bonanomi, Julio C. Orellana, Grazia Fazio, Fred H. Rodriguez, III, Loreani P. Noguera, Claire Booth, Valentina Jarur-Chamy, Marissa Shams, Maria Iascone, Maja Vukic, Serena Gasperini, Manuel Quadri, Amairelys Barroeta Seijas, Elizabeth Rivers, Mario Mauri, Raffaele Badolato, Gianni Cazzaniga, Cristina Bugarin, Giuseppe Gaipa, Wilma G. M. Kroes, Daniele Moratto, Monique M. van Oostaijen-ten Dam, Frank Baas, Silvère van der Maarel, Rocco Piazza, Zeynep H. Coban-Akdemir, James R. Lupski, Bo Yuan, Ivan K. Chinn, Lucia Daxinger, Andrea Biondi
Journal: Blood
Blood (2021) 137 (4): 493–499.
https://doi.org/10.1182/blood.2020006441
Published: 2021
Includes: Supplemental data
Journal Articles

Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102

Agnieszka Czechowicz, MDPhD, Rajni Agarwal, MD, Julián Sevilla, MD PhD, Paula Río, PhD, Susana Navarro, PhD, Brian C Beard, PhD, Kenneth M Law, PhD, Grace Choi, BS, Miriam Zeini, PhD, Eileen Nicoletti, MD, John E Wagner, MD, Claire Booth, MBBS, PhD MSc, Jonathan D Schwartz, MD, Juan A Bueren, PhD, Maria Grazia Roncarolo, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 14.
https://doi.org/10.1182/blood-2020-137106
Published: 2020
Journal Articles

Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress - NCT04201405

Jane L Kinsella, MBChB, Robert F Wynn, MRCP,MDFRCPath, Brian Bigger, PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Claire Booth, MBBS, PhD MSc, Karen Buckland, PhD, Natalia Izotova, Stewart Rust, B.Sc. (Hons), D.Clin.Psych, Pg.Dip., C.Psychol., AfBPS., Daniel Weisberg, BSc, PhD PGCert, ClinPsyD, Heather J Church, PhD, Kathryn L Brammeier, Karen L Tylee, Ceri Jones, BSc, Helena Lee, Laura Ford, Atusa Sadegholnejat, Stuart Ellison, Simon A Jones, MBChB, BSc, MRCPCH
Journal: Blood
Blood (2020) 136 (Supplement 1): 15–16.
https://doi.org/10.1182/blood-2020-141762
Published: 2020
Journal Articles

Bipotent Lymphoid Progenitors Independently Maintain Long-Term Genetically Engineered T and NK Cell Production in Humans

Natalia Izotova, Christine Rivat, Cristina Baricordi, PhD, Elena Blanco-Alvarez, Danilo Pellin, Eleanor Watt, Soragia Athina Gkazi, PhD, Stuart Adams, PhD, Kimberly Gilmour, Jinhua Xu Bayford, Claire Booth, MBBS, PhD MSc, Bobby H Gaspar, MBBS, PhD BS, Adrian J. Thrasher, MBBS, PhD FMedSci, Luca Biasco, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 50.
https://doi.org/10.1182/blood-2020-137001
Published: 2020
Journal Articles

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Donald B. Kohn, MD, Kit L. Shaw, PhD, Elizabeth Garabedian, RN, MS, Denise Ann Carbonaro-Sarracino, PhD, Theodore B. Moore, MD, Satiro N. De Oliveira, MD, Gay M. Crooks, MD, John Tse, Sally Shupien, Dayna Terrazas, Alejandra Davila, Amalia Icreverzi, PhD, Allen Yu, Krista M. Chun, Christian E. Casas, Provaboti Barman, PhD, Maritess Coronel, Beatriz Campo Fernandez, PhD, Ruixue Zhang, Roger P. Hollis, PhD, Chilenwa Uzowuru, Hilory Ricketts, Jinhua Xu Bayford, Valentina Trevisan, Serena Arduini, PhD, Frances Lynn, Mahesh Kudari, MBBS, MA, Andrea Spezzi, MD MBBS, Lilith Reeves, MS, MT(ASCP), Kenneth Cornetta, Robert A. Sokolic, MD, Roberta Parrott, BS, Rebecca Buckley, MD, Claire Booth, Fabio Candotti, MD, Harry L. Malech, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, H. Bobby Gaspar, MBBS, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3345.
https://doi.org/10.1182/blood-2019-123432
Published: 2019
Journal Articles

Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis

Giovanna Lucchini, Rebecca Marsh, Kimberly Gilmour, Austen Worth, Zohreh Nademi, Anupama Rao, Claire Booth, Persis Amrolia, Juliana Silva, Robert Chiesa, Robert Wynn, Kai Lehmberg, Itziar Astigarraga, Tayfun Güngör, Jan Stary, Despina Moshous, Marianne Ifversen, Daniel Zinn, Michael Jordan, Ashish Kumar, Takahiro Yasumi, Paul Veys, Kanchan Rao
Journal: Blood
Blood (2018) 132 (19): 2088–2096.
https://doi.org/10.1182/blood-2018-01-827485
Published: 2018
Includes: Supplemental data