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Journal Articles

Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues

Frank W. G. Leebeek, Wolfgang Miesbach
Journal: Blood
Blood (2021) 138 (11): 923–931.
https://doi.org/10.1182/blood.2019003777
Published: 2021
Journal Articles

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Catherine M. Bollard
Journal: Blood
Blood (2021) 138 (11): 913.
https://doi.org/10.1182/blood.2021012891
Published: 2021
Journal Articles

Gene therapy for sickle cell disease: moving from the bench to the bedside

Allistair A. Abraham, John F. Tisdale
Journal: Blood
Blood (2021) 138 (11): 932–941.
https://doi.org/10.1182/blood.2019003776
Published: 2021
Journal Articles

Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

Clinical Trials & Observations
Richard J. Jones, Michael R. DeBaun
Journal: Blood
Blood (2021) 138 (11): 942–947.
https://doi.org/10.1182/blood.2021011488
Published: 2021
Journal Articles

A new “FIX” for hemophilia B gene therapy

Graça Almeida-Porada
Journal: Blood
Blood (2021) 137 (21): 2860–2861.
https://doi.org/10.1182/blood.2021011753
Published: 2021
Journal Articles

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Brief Report
Nisha Nair, Dries De Wolf, Phuong Anh Nguyen, Quang Hong Pham, Ermira Samara-Kuko, Jeff Landau, Grant E. Blouse, Marinee K. Chuah, Thierry VandenDriessche
Journal: Blood
Blood (2021) 137 (21): 2902–2906.
https://doi.org/10.1182/blood.2020006005
Published: 2021
Includes: Supplemental data
Journal Articles

von Willebrand disease: what does the future hold?

Cécile V. Denis, Sophie Susen, Peter J. Lenting
Journal: Blood
Blood (2021) 137 (17): 2299–2306.
https://doi.org/10.1182/blood.2020008501
Published: 2021
Journal Articles

No CpGs for AAVs?

Lindsey A. George
Journal: Blood
Blood (2021) 137 (6): 721–723.
https://doi.org/10.1182/blood.2020009285
Published: 2021
Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Clinical Trials & Observations
Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan
Journal: Blood
Blood (2021) 137 (6): 763–774.
https://doi.org/10.1182/blood.2019004625
Published: 2021
Includes: Supplemental data
Journal Articles

Diamond-Blackfan anemia

Lydie Da Costa, Thierry Leblanc, Narla Mohandas
Journal: Blood
Blood (2020) 136 (11): 1262–1273.
https://doi.org/10.1182/blood.2019000947
Published: 2020
Journal Articles

Natural gene therapy in hematopoietic disorders: GATA too

Marjolijn C. Jongmans, Roland P. Kuiper
Journal: Blood
Blood (2020) 136 (8): 923–924.
https://doi.org/10.1182/blood.2020007001
Published: 2020
Journal Articles

Factor VIII exhibits chaperone-dependent and glucose-regulated reversible amyloid formation in the endoplasmic reticulum

Juthakorn Poothong, Anita Pottekat, Marina Siirin, Alexandre Rosa Campos, Adrienne W. Paton, James C. Paton, Jacqueline Lagunas-Acosta, Zhouji Chen, Mark Swift, Niels Volkmann, Dorit Hanein, Jing Yong, Randal J. Kaufman
Journal: Blood
Blood (2020) 135 (21): 1899–1911.
https://doi.org/10.1182/blood.2019002867
Published: 2020
Includes: Supplemental data
Journal Articles

The evolution of viral integration site analysis

Stefan Radtke, Hans-Peter Kiem
Journal: Blood
Blood (2020) 135 (15): 1192–1193.
https://doi.org/10.1182/blood.2020005115
Published: 2020
Journal Articles

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Emmanuelle Six, Agathe Guilloux, Adeline Denis, Arnaud Lecoules, Alessandra Magnani, Romain Vilette, Frances Male, Nicolas Cagnard, Marianne Delville, Elisa Magrin, Laure Caccavelli, Cécile Roudaut, Clemence Plantier, Steicy Sobrino, John Gregg, Christopher L. Nobles, John K. Everett, Salima Hacein-Bey-Abina, Anne Galy, Alain Fischer, Adrian J. Thrasher, Isabelle André, Marina Cavazzana, Frederic D. Bushman
Journal: Blood
Blood (2020) 135 (15): 1219–1231.
https://doi.org/10.1182/blood.2019002350
Published: 2020
Includes: Supplemental data
Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD
Journal: Blood
Blood (2019) 134 (Supplement_2): LBA-5.
https://doi.org/10.1182/blood-2019-132745
Published: 2019
Journal Articles

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Valentina Ghiaccio, PhD, Alisa Dong, PhD, Irene Motta, MD, Shuling Guo, PhD, Raechel Peralta, MS, Susan M Freier, Andy Watt, Yasuhiro Ikawa, MD PhD, Maxwell Chappell, MS, Coralea Stephanou, PhD, Paola Delbini, PhD, Connie Chen, Soteroula Christou, MD, Marina Kleanthous, PhD, Kim Smith-Whitley, MD, Deepa Manwani, MD, Carla Casu, PhD, Maria Domenica Cappellini, MD, Osheiza Abdulmalik, DVM, Stefano Rivella, PhD, Laura Breda, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2244.
https://doi.org/10.1182/blood-2019-127017
Published: 2019
Journal Articles

Allogeneic Hematopoietic Stem Cell Transplant Versus Gene Therapy in Sickle Cell Disease: A Systematic Review

Lianne E Rotin, MD PhD, Auro Viswabandya, MD, Rajat Kumar, MD, Kevin H. M. Kuo, MDMSc,FRCPC
Journal: Blood
Blood (2019) 134 (Supplement_1): 4714.
https://doi.org/10.1182/blood-2019-124989
Published: 2019
Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Roxane Labrosse, MD FRCPC, Julia Chu, MD MPH, Myriam Armant, PhD, Jet van der Spek, MD, Alexandra Miggelbrink, MD, Johnson Fong, John K. Everett, PhD, Hayley Raymond, BA, Lyanna Kessler, Colleen Dansereau, MSN, RN, CPN, Brenda Mackinnon, BSN, RN, Stephanie Koo, Emily Morris, Wendy B. London, PhD, Ahmet Ozen, MD, Safa Baris, MD, Jenny M. Despotovic, DO, MS, Lisa Forbes, MD, Akihiko Saitoh, MD, Takayuki Takachi, MD, Alejandra King, MD, Thi Mai Anh Thi Nguyen, MD, Vy Do Uyen Vu, MD, Frederic D. Bushman, PhD, Anne Galy, PharmD, PhD, Luigi Notarangelo, MD, David A Williams, MD, Sung-Yun Pai, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4629.
https://doi.org/10.1182/blood-2019-126161
Published: 2019
Journal Articles

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Paula Rio, PhD, Susana Navarro, PhD, Rebeca Sanchez-Dominguez, PhD, Jose C Segovia, PhD, Wei Wang, PhD, Eva Merino, Rosa M Yañez, PhD, Jose Antonio Casado, PhD, Yari A. Gimenez, Francisco J Roman-Rodriguez, PhD, Omaira Alberquilla, Eva Galvez, MD, Raquel Hladun, MD, Jordi Barquinero, PhD, Anne Galy, PharmD, PhD, Nagore García de Andoín, MD, Ricardo López, MD PhD, Albert Catalá, MD PhD, Marina Cavazzana, Francois Lefrere, MD, Thierry Leblanc, MD, Jonathan D Schwartz, MD, Roser Pujol, PhD, Jordi Surrallés, PhD, Jean Soulier, MD PhD, Manfred Schmidt, PhD, Cristina Díaz de Heredia, MD PhD, Julian Sevilla, MD PhD, Juan A. Bueren, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4627.
https://doi.org/10.1182/blood-2019-129686
Published: 2019
Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4628.
https://doi.org/10.1182/blood-2019-125807
Published: 2019