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Journal Articles

Wild-type HIV infection after treatment with lentiviral gene therapy for β-thalassemia

Suradej Hongeng, Usanarat Anurathapan, Duantida Songdej, Angsana Phuphuakrat, Kesinee Jongrak, Geoffrey Parsons, Briana Deary, Melissa Bonner, Gabor Veres, Mohammed Asmal
Journal: Blood Advances
Blood Adv (2021) 5 (13): 2701–2706.
https://doi.org/10.1182/bloodadvances.2020003680
Published: 2021
Journal Articles

Molecular analysis of the erythroid phenotype of a patient with BCL11A haploinsufficiency

Clinical Trials & Observations
Marja W. Wessels, Marjon H. Cnossen, Thamar B. van Dijk, Nynke Gillemans, K. L. Juliëtte Schmidt, Kirsten van Lom, Divya S. Vinjamur, Steven Coyne, Ryo Kurita, Yukio Nakamura, Stella A. de Man, Rolph Pfundt, Zakia Azmani, Rutger W. W. Brouwer, Daniel E. Bauer, Mirjam C. G. N. van den Hout, Wilfred F. J. van IJcken, Sjaak Philipsen
Journal: Blood Advances
Blood Adv (2021) 5 (9): 2339–2349.
https://doi.org/10.1182/bloodadvances.2020003753
Published: 2021
Includes: Supplemental data
Journal Articles

MetAP2 inhibition modifies hemoglobin S to delay polymerization and improves blood flow in sickle cell disease

Melanie Demers, Sarah Sturtevant, Kevin R. Guertin, Dipti Gupta, Kunal Desai, Benjamin F. Vieira, Wenjing Li, Alexandra Hicks, Ayman Ismail, Bronner P. Gonçalves, Giuseppe Di Caprio, Ethan Schonbrun, Scott Hansen, Faik N. Musayev, Martin K. Safo, David K. Wood, John M. Higgins, David R. Light
Journal: Blood Advances
Blood Adv (2021) 5 (5): 1388–1402.
https://doi.org/10.1182/bloodadvances.2020003670
Published: 2021
Includes: Supplemental data
Journal Articles

Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice

Chang Li, Kevin A. Goncalves, Tamás Raskó, Amit Pande, Sucheol Gil, Zhinan Liu, Zsuzsanna Izsvák, Thalia Papayannopoulou, John C. Davis, Jr, Hans-Peter Kiem, André Lieber
Journal: Blood Advances
Blood Adv (2021) 5 (5): 1239–1249.
https://doi.org/10.1182/bloodadvances.2020003714
Published: 2021
Includes: Supplemental data
Journal Articles

Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells

Giulia Pavani, Anna Fabiano, Marine Laurent, Fatima Amor, Erika Cantelli, Anne Chalumeau, Giulia Maule, Alexandra Tachtsidi, Jean-Paul Concordet, Anna Cereseto, Fulvio Mavilio, Giuliana Ferrari, Annarita Miccio, Mario Amendola
Journal: Blood Advances
Blood Adv (2021) 5 (5): 1137–1153.
https://doi.org/10.1182/bloodadvances.2020001996
Published: 2021
Includes: Supplemental data
Journal Articles

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

Chang Li, Aphrodite Georgakopoulou, Arpit Mishra, Sucheol Gil, R. David Hawkins, Evangelia Yannaki, André Lieber
Journal: Blood Advances
Blood Adv (2021) 5 (4): 1122–1135.
https://doi.org/10.1182/bloodadvances.2020003702
Published: 2021
Includes: Supplemental data
Journal Articles

Zhan J, Irudayam MJ, Nakamura Y, Kurita R, Nienhuis AW. High level of fetal-globin reactivation by designed transcriptional activator-like effector. Blood Adv. 2020;4(4):687-695.

Journal: Blood Advances
Blood Adv (2020) 4 (7): 1197.
https://doi.org/10.1182/bloodadvances.2020001766
Published: 2020
Journal Articles

High level of fetal-globin reactivation by designed transcriptional activator-like effector

Jun Zhan, Maria Johnson Irudayam, Yukio Nakamura, Ryo Kurita, Arthur W. Nienhuis
Journal: Blood Advances
Blood Adv (2020) 4 (4): 687–695.
https://doi.org/10.1182/bloodadvances.2019000482
Published: 2020
Includes: Supplemental data
Journal Articles

Genome editing of HBG1 and HBG2 to induce fetal hemoglobin

Jean-Yves Métais, Phillip A. Doerfler, Thiyagaraj Mayuranathan, Daniel E. Bauer, Stephanie C. Fowler, Matthew M. Hsieh, Varun Katta, Sagar Keriwala, Cicera R. Lazzarotto, Kevin Luk, Michael D. Neel, S. Scott Perry, Samuel T. Peters, Shaina N. Porter, Byoung Y. Ryu, Akshay Sharma, Devlin Shea, John F. Tisdale, Naoya Uchida, Scot A. Wolfe, Kaitly J. Woodard, Yuxuan Wu, Yu Yao, Jing Zeng, Shondra Pruett-Miller, Shengdar Q. Tsai, Mitchell J. Weiss
Journal: Blood Advances
Blood Adv (2019) 3 (21): 3379–3392.
https://doi.org/10.1182/bloodadvances.2019000820
Published: 2019
Includes: Supplemental data
Journal Articles

BCL2L1 is associated with γ-globin gene expression

Yan Dai, Elmutaz M. Shaikho, Jessica Perez, Carolyn A. Wilson, Lesley Y. Liu, Mitchell R. White, John J. Farrell, David H. K. Chui, Paola Sebastiani, Martin H. Steinberg
Journal: Blood Advances
Blood Adv (2019) 3 (20): 2995–3001.
https://doi.org/10.1182/bloodadvances.2019032243
Published: 2019
Journal Articles

High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells

Hongjie Wang, Zhinan Liu, Chang Li, Sucheol Gil, Thalia Papayannopoulou, Christopher B. Doering, André Lieber
Journal: Blood Advances
Blood Adv (2019) 3 (19): 2883–2894.
https://doi.org/10.1182/bloodadvances.2019000706
Published: 2019
Includes: Supplemental data
Journal Articles

Decoding the role of SOD2 in sickle cell disease

Atinuke M. Dosunmu-Ogunbi, Katherine C. Wood, Enrico M. Novelli, Adam C. Straub
Journal: Blood Advances
Blood Adv (2019) 3 (17): 2679–2687.
https://doi.org/10.1182/bloodadvances.2019000527
Published: 2019
Journal Articles

The E3 ligase adaptor molecule SPOP regulates fetal hemoglobin levels in adult erythroid cells

Xianjiang Lan, Eugene Khandros, Peng Huang, Scott A. Peslak, Saurabh K. Bhardwaj, Jeremy D. Grevet, Osheiza Abdulmalik, Hongxin Wang, Cheryl A. Keller, Belinda Giardine, Josue Baeza, Emily R. Duffner, Osama El Demerdash, Xiaoli S. Wu, Christopher R. Vakoc, Benjamin A. Garcia, Ross C. Hardison, Junwei Shi, Gerd A. Blobel
Journal: Blood Advances
Blood Adv (2019) 3 (10): 1586–1597.
https://doi.org/10.1182/bloodadvances.2019032318
Published: 2019
Includes: Supplemental data
Journal Articles

Prevalence of inherited blood disorders and associations with malaria and anemia in Malawian children

Global Advances
Patrick T. McGann, Anne M. Williams, Graham Ellis, Kathryn E. McElhinney, Laurel Romano, Julia Woodall, Thad A. Howard, Gerald Tegha, Robert Krysiak, R. Murray Lark, E. Louise Ander, Carine Mapango, Kenneth I. Ataga, Satish Gopal, Nigel S. Key, Russell E. Ware, Parminder S. Suchdev
Journal: Blood Advances
Blood Adv (2018) 2 (21): 3035–3044.
https://doi.org/10.1182/bloodadvances.2018023069
Published: 2018
Journal Articles

Induced pluripotent stem cell–based mapping of β-globin expression throughout human erythropoietic development

Kim Vanuytsel, Taylor Matte, Amy Leung, Zaw Htut Naing, Tasha Morrison, David H. K. Chui, Martin H. Steinberg, George J. Murphy
Journal: Blood Advances
Blood Adv (2018) 2 (15): 1998–2011.
https://doi.org/10.1182/bloodadvances.2018020560
Published: 2018
Includes: Supplemental data
Journal Articles

Biological impact of α genes, β haplotypes, and G6PD activity in sickle cell anemia at baseline and with hydroxyurea

Clinical Trials & Observations Global Advances
Françoise Bernaudin, Cécile Arnaud, Annie Kamdem, Isabelle Hau, Françoise Lelong, Ralph Epaud, Corinne Pondarré, Serge Pissard
Journal: Blood Advances
Blood Adv (2018) 2 (6): 626–637.
https://doi.org/10.1182/bloodadvances.2017014555
Published: 2018
Journal Articles

Allogeneic bone marrow transplant in the absence of cytoreductive conditioning rescues mice with β-thalassemia major

Yongliang Huo, Jonathan R. Lockhart, Shanrun Liu, Suean Fontenard, Mike Berlett, Thomas M. Ryan
Journal: Blood Advances
Blood Adv (2017) 1 (25): 2421–2432.
https://doi.org/10.1182/bloodadvances.2017009449
Published: 2017
Includes: Supplemental data
Journal Articles

KLF1 directly activates expression of the novel fetal globin repressor ZBTB7A/LRF in erythroid cells

Laura J. Norton, Alister P. W. Funnell, Jon Burdach, Beeke Wienert, Ryo Kurita, Yukio Nakamura, Sjaak Philipsen, Richard C. M. Pearson, Kate G. R. Quinlan, Merlin Crossley
Journal: Blood Advances
Blood Adv (2017) 1 (11): 685–692.
https://doi.org/10.1182/bloodadvances.2016002303
Published: 2017
Includes: Supplemental data
Journal Articles

Hemoglobin variants identified in the Uganda Sickle Surveillance Study

Global Advances
Beverly A. Schaefer, Charles Kiyaga, Thad A. Howard, Grace Ndeezi, Arielle G. Hernandez, Isaac Ssewanyana, Mary C. Paniagua, Christopher M. Ndugwa, Jane R. Aceng, Russell E. Ware
Journal: Blood Advances
Blood Adv (2016) 1 (1): 93–100.
https://doi.org/10.1182/bloodadvances.2016000950
Published: 2016
Includes: Supplemental data