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Journal Articles

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

Allistair A. Abraham, John F. Tisdale

Blood (2021) 138 (11): 932–941.

https://doi.org/10.1182/blood.2019003776

Published: 2021

Journal Articles

No CpGs for AAVs?

Lindsey A. George

Blood (2021) 137 (6): 721–723.

https://doi.org/10.1182/blood.2020009285

Published: 2021

Journal Articles

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Blood (2019) 134 (Supplement_1): 608.

https://doi.org/10.1182/blood-2019-127439

Published: 2019

Journal Articles

MDSC Suppression of CAR T Cells Can be Reduced By Targeted Signaling Disruption

Estelle V Cervantes, Justin C. Boucher, PhD, Sae Bom Lee, Kristen Spitler, Kayla Reid, Marco L. Davila, MD PhD

Blood (2019) 134 (Supplement_1): 4438.

https://doi.org/10.1182/blood-2019-122752

Published: 2019

Journal Articles

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

Lian Yu, Wei Hao Wu, WANG Shunqing, Ken H. Young, Funeng Jiang, PhD

Blood (2019) 134 (Supplement_1): 5754.

https://doi.org/10.1182/blood-2019-129931

Published: 2019

Journal Articles

Novel Cellular Immunotherapy Using Allogeneic Vγ9/δ2-T Cells Gene-Modified to Express HTLV-1 P40Tax-Specific TCR for the Treatment of Adult T Cell Leukemia

Hiroshi Fujiwara, MD PhD, Satoshi Okumura, Yoshihiro Miyahara, Linan Wan, Isao Tawara, MD PhD, Tatsuro Jo, MD PhD, Yuetsu Tanaka, PhD, Yoshimasa Tanaka, PhD, Hiroaki Ikeda, Hiroshi Shiku

Blood (2019) 134 (Supplement_1): 3216.

https://doi.org/10.1182/blood-2019-122412

Published: 2019

Journal Articles

Rapid Personalized Manufacture (RPM) of Sleeping Beauty System-Generated NY-ESO-1-Specific TCR-T Cells Co-Expressing Membrane-Bound IL-15 Yields Anti-Tumor Responses

Lenka V Hurton, PhD, Tiejuan Mi, Kirsten C Switzer, PhD, Ling Zhang, Cuiping Dai, Misun Kim, Karishma R Bavisi, Matthew J Figliola, Vazquez M Lydia, Sourindra N Maiti, Shihuang Su, Drew C Deniger, PhD, Simon Olivares, Laurence J N Cooper, MD PhD, Harjeet Singh, MSc, PhD

Blood (2019) 134 (Supplement_1): 3218.

https://doi.org/10.1182/blood-2019-131419

Published: 2019

Journal Articles

TP73 As Novel Determinant of Resistance to BCL-2 Inhibition in Acute Myeloid Leukemia

Yuki Nishida, MD PhD, Jo Ishizawa, MD PhD, Vivian Ruvolo, MS, Feng Wang, PhD, Koichi Takahashi, MD, Po Yee Mak, Bing Z Carter, PhD, Michael Andreeff, MD PhD

Blood (2019) 134 (Supplement_1): 1251.

https://doi.org/10.1182/blood-2019-127182

Published: 2019

Journal Articles

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Steven Pipe, MD, Michael Becka, Elke Detering, Konstantina Vanevski, Toshko Lissitchkov

Blood (2019) 134 (Supplement_1): 4630.

https://doi.org/10.1182/blood-2019-125764

Published: 2019

Journal Articles

In Vivo hemostatic Significance of Activated Protein C in Factor VIIIa Regulation

Amelia R. Wilhelm, B.S., Nicole A. Parsons, B.S., Charles T. Esmon, PhD, Rodney M. Camire, PhD, Lindsey A. George, MD

Blood (2019) 134 (Supplement_1): 93.

https://doi.org/10.1182/blood-2019-124493

Published: 2019

Journal Articles

Preclinical Assessment of Non-Virally Engineered CD33.CAR Cytokine-Induced Killer (CIK) Cells in Chemoresistant AML Patient-Derived Xenografts

Maria Caterina Rotiroti, PhD, Chiara Buracchi, PhD, Silvia Arcangeli, PhD, Chiara F Magnani, PhD, Claudia Cappuzzello, PhD, Zsuzsanna Izsvak, PhD, Stefania Galimberti, PhD, Maria Grazia Valsecchi, PhD, Giuseppe Dastoli, MD, Marta Serafini, PhD, Ettore Biagi, MD, Andrea Biondi, MD, Sarah Tettamanti, PhD

Blood (2019) 134 (Supplement_1): 2665.

https://doi.org/10.1182/blood-2019-130399

Published: 2019

Journal Articles

Preclinical Characterization of Prgn-3006 Ultracar-T™ for the Treatment of AML and MDS: Non-Viral, Multigenic Autologous CAR-T Cells Administered One Day after Gene Transfer

Tim Chan, PhD, Xiaohong Ma, Fernando Carvajal-Borda, DVM, Jose Velez, Jacques Plummer, Lindsey Shepard, Rutul R Shah, David A Sallman, MD, Helen Sabzevari, PhD

Blood (2019) 134 (Supplement_1): 2660.

https://doi.org/10.1182/blood-2019-130617

Published: 2019

Journal Articles

Anti-Apoptotic BCL-2 Family Members Confer Resistance to Calicheamicin-Based Antibody-Drug Conjugate Therapy of Acute Leukemia

Colin D. Godwin, MDMPhil, Olivia M. Bates, BA, Sae Rin Jean, PhD, George S. Laszlo, PhD, Eliotte E. Garling, BA, Mary E. Beddoe, BS, Michael H. Cardone, PhD, Roland B. Walter, MD PhD MS

Blood (2019) 134 (Supplement_1): 2561.

https://doi.org/10.1182/blood-2019-126110

Published: 2019

Journal Articles

Foamy Virus Based Vectors for CAR-T Cell Development

Emmanouil Simantirakis, MSc, Vassilis Atsaves, PhD, Ioannis Tsironis, BSc MSc, Margarita Gkyzi, BSc, Kostas Konstantopoulos, MD PhD, George Vassilopoulos, MD PhD

Blood (2019) 134 (Supplement_1): 4646.

https://doi.org/10.1182/blood-2019-123022

Published: 2019

Journal Articles

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Julie Kanter, MD, John F. Tisdale, MD, Markus Y. Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Alexis A. Thompson, MD MPH, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 990.

https://doi.org/10.1182/blood-2019-128894

Published: 2019

Journal Articles

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Steven Pipe, MD, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MBChB, FRCP, Susan U Lattimore, Frank Leebeek, MD PhD, Wolfgang Miesbach, MD PhD, Michael Recht, MD PhD, Esteban Gomez, MD, Alison Long, Robert Gut, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 3348.

https://doi.org/10.1182/blood-2019-128765

Published: 2019

Journal Articles

Seroprevalence of Pre-Existing Nabs Against AAV1, 2, 5, 6 and 8 in South African Hemophilia B Patient Population

Anna Majowicz, PhD, Nolukholo Ncete, MBBCH, Floris van Waes, MSc, Nikki Timmer, BSc, Sander J van Deventer, MD PhD, Johnny N Mahlangu, MBBCH, MMed, FCPath, Valerie Ferreira, PhD

Blood (2019) 134 (Supplement_1): 3353.

https://doi.org/10.1182/blood-2019-128217

Published: 2019

Journal Articles

Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia

Kavitha Rajavel, Mila Ayash-Rashkovsky, Ying Tang, Bagirath Gangadharan, Maurus de la Rosa, Bruce Ewenstein

Blood (2019) 134 (Supplement_1): 3349.

https://doi.org/10.1182/blood-2019-123666

Published: 2019

Journal Articles

Megakaryocytes and platelets from a novel human adipose tissue–derived mesenchymal stem cell line

Keiichi Tozawa, Yukako Ono-Uruga, Masaki Yazawa, Taisuke Mori, Mitsuru Murata, Shinichiro Okamoto, Yasuo Ikeda, Yumiko Matsubara

Blood (2019) 133 (7): 633–643.

https://doi.org/10.1182/blood-2018-04-842641

Published: 2019

Includes: Supplemental data

Journal Articles

Update on clinical gene therapy for hemophilia

George Q. Perrin, Roland W. Herzog, David M. Markusic

Blood (2019) 133 (5): 407–414.

https://doi.org/10.1182/blood-2018-07-820720

Published: 2019

NARROW

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

No CpGs for AAVs?

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

MDSC Suppression of CAR T Cells Can be Reduced By Targeted Signaling Disruption

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

Novel Cellular Immunotherapy Using Allogeneic Vγ9/δ2-T Cells Gene-Modified to Express HTLV-1 P40Tax-Specific TCR for the Treatment of Adult T Cell Leukemia

Rapid Personalized Manufacture (RPM) of Sleeping Beauty System-Generated NY-ESO-1-Specific TCR-T Cells Co-Expressing Membrane-Bound IL-15 Yields Anti-Tumor Responses

TP73 As Novel Determinant of Resistance to BCL-2 Inhibition in Acute Myeloid Leukemia

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

In Vivo hemostatic Significance of Activated Protein C in Factor VIIIa Regulation

Preclinical Assessment of Non-Virally Engineered CD33.CAR Cytokine-Induced Killer (CIK) Cells in Chemoresistant AML Patient-Derived Xenografts

Preclinical Characterization of Prgn-3006 Ultracar-T™ for the Treatment of AML and MDS: Non-Viral, Multigenic Autologous CAR-T Cells Administered One Day after Gene Transfer

Anti-Apoptotic BCL-2 Family Members Confer Resistance to Calicheamicin-Based Antibody-Drug Conjugate Therapy of Acute Leukemia

Foamy Virus Based Vectors for CAR-T Cell Development

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Seroprevalence of Pre-Existing Nabs Against AAV1, 2, 5, 6 and 8 in South African Hemophilia B Patient Population

Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia

Megakaryocytes and platelets from a novel human adipose tissue–derived mesenchymal stem cell line

Update on clinical gene therapy for hemophilia

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American Society of Hematology

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American Society of Hematology

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