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1362.
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A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition. [electronic resource] by
- Traxler, Elizabeth A
- Yao, Yu
- Wang, Yong-Dong
- Woodard, Kaitly J
- Kurita, Ryo
- Nakamura, Yukio
- Hughes, Jim R
- Hardison, Ross C
- Blobel, Gerd A
- Li, Chunliang
- Weiss, Mitchell J
Producer: 20170810
In:
Nature medicine vol. 22
Availability: No items available.
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1363.
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1364.
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1365.
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Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease. [electronic resource] by
- Park, So Hyun
- Lee, Ciaran M
- Dever, Daniel P
- Davis, Timothy H
- Camarena, Joab
- Srifa, Waracharee
- Zhang, Yankai
- Paikari, Alireza
- Chang, Alicia K
- Porteus, Matthew H
- Sheehan, Vivien A
- Bao, Gang
Producer: 20191209
In:
Nucleic acids research vol. 47
Availability: No items available.
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1366.
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Development of K562 cell clones expressing beta-globin mRNA carrying the beta039 thalassaemia mutation for the screening of correctors of stop-codon mutations. [electronic resource] by
- Salvatori, Francesca
- Cantale, Vera
- Breveglieri, Giulia
- Zuccato, Cristina
- Finotti, Alessia
- Bianchi, Nicoletta
- Borgatti, Monica
- Feriotto, Giordana
- Destro, Federica
- Canella, Alessandro
- Breda, Laura
- Rivella, Stefano
- Gambari, Roberto
Producer: 20090914
In:
Biotechnology and applied biochemistry vol. 54
Availability: No items available.
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1367.
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1368.
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Safe mobilization of CD34+ cells in adults with β-thalassemia and validation of effective globin gene transfer for clinical investigation. [electronic resource] by
- Boulad, Farid
- Wang, Xiuyan
- Qu, Jinrong
- Taylor, Clare
- Ferro, Leda
- Karponi, Garyfalia
- Bartido, Shirley
- Giardina, Patricia
- Heller, Glenn
- Prockop, Susan E
- Maggio, Aurelio
- Sadelain, Michel
- Rivière, Isabelle
Producer: 20140509
In:
Blood vol. 123
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1371.
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1372.
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CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells. [electronic resource] by
- Hoban, Megan D
- Lumaquin, Dianne
- Kuo, Caroline Y
- Romero, Zulema
- Long, Joseph
- Ho, Michelle
- Young, Courtney S
- Mojadidi, Michelle
- Fitz-Gibbon, Sorel
- Cooper, Aaron R
- Lill, Georgia R
- Urbinati, Fabrizia
- Campo-Fernandez, Beatriz
- Bjurstrom, Carmen F
- Pellegrini, Matteo
- Hollis, Roger P
- Kohn, Donald B
Producer: 20170606
In:
Molecular therapy : the journal of the American Society of Gene Therapy vol. 24
Availability: No items available.
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1374.
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Direct Promoter Repression by BCL11A Controls the Fetal to Adult Hemoglobin Switch. [electronic resource] by
- Liu, Nan
- Hargreaves, Victoria V
- Zhu, Qian
- Kurland, Jesse V
- Hong, Jiyoung
- Kim, Woojin
- Sher, Falak
- Macias-Trevino, Claudio
- Rogers, Julia M
- Kurita, Ryo
- Nakamura, Yukio
- Yuan, Guo-Cheng
- Bauer, Daniel E
- Xu, Jian
- Bulyk, Martha L
- Orkin, Stuart H
Producer: 20190208
In:
Cell vol. 173
Availability: No items available.
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