Treating sickle cell anemia

被引：51

作者：

Tisdale, John F. ^{[1
]}

Thein, Swee Lay ^{[2
]}

Eaton, William A. ^{[3
]}

机构：

[1] NHLBI, Cellular & Mol Therapeut Branch, NIH, Bldg 10, Bethesda, MD 20892 USA

[2] NHLBI, Sickle Cell Branch, NIH, Bldg 10, Bethesda, MD 20892 USA

[3] NIDDK, Lab Chem Phys, NIH, Bethesda, MD 20892 USA

来源：

SCIENCE | 2020年 / 367卷 / 6483期

基金：

美国国家卫生研究院;

关键词：

DISEASE;

D O I：

10.1126/science.aba3827

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

引用

页码：1198 / 1199

页数：2

共 15 条

[1] Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease [J].

Ataga, K. I. ;

Kutlar, A. ;

Kanter, J. ;

Liles, D. ;

Cancado, R. ;

Friedrisch, J. ;

Guthrie, T. H. ;

Knight-Madden, J. ;

Alvarez, O. A. ;

Gordeuk, V. R. ;

Gualandro, S. ;

Colella, M. P. ;

Smith, W. R. ;

Rollins, S. A. ;

Stocker, J. W. ;

Rother, R. P. .

NEW ENGLAND JOURNAL OF MEDICINE, 2017, 376 (05) :429-439

[2] CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells [J].

Dever, Daniel P. ;

Bak, Rasmus O. ;

Reinisch, Andreas ;

Camarena, Joab ;

Washington, Gabriel ;

Nicolas, Carmencita E. ;

Pavel-Dinu, Mara ;

Saxena, Nivi ;

Wilkens, Alec B. ;

Mantri, Sruthi ;

Uchida, Nobuko ;

Hendel, Ayal ;

Narla, Anupama ;

Majeti, Ravindra ;

Weinberg, Kenneth I. ;

Porteus, Matthew H. .

NATURE, 2016, 539 (7629) :384-389

[3] Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study [J].

Eapen, Mary ;

Brazauskas, Ruta ;

Walters, Mark C. ;

Bernaudin, Francoise ;

Bo-Subait, Khalid ;

Fitzhugh, Courtney D. ;

Hankins, Jane S. ;

Kanter, Julie ;

Meerpohl, Joerg J. ;

Bolanos-Meade, Javier ;

Panepinto, Julie A. ;

Rondelli, Damiano ;

Shenoy, Shalini ;

Williamson, Joi ;

Woolford, Teonna L. ;

Gluckman, Eliane ;

Wagner, John E. ;

Tisdale, John F. .

LANCET HAEMATOLOGY, 2019, 6 (11) :E585-E596

[4] Linus Pauling and sickle cell disease [J].

Eaton, WA .

BIOPHYSICAL CHEMISTRY, 2003, 100 (1-3) :109-116

[5] Treating sickle cell disease by targeting HbS polymerization [J].

Eaton, William A. ;

Bunn, H. Franklin .

BLOOD, 2017, 129 (20) :2719-2726

[6] Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies [J].

Magrin, Elisa ;

Miccio, Annarita ;

Cavazzana, Marina .

BLOOD, 2019, 134 (15) :1203-1213

[7] Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin [J].

May, C ;

Rivella, S ;

Callegari, J ;

Heller, G ;

Gaensler, KML ;

Luzzatto, L ;

Sadelain, M .

NATURE, 2000, 406 (6791) :82-86

[8] A QTL influencing F cell production maps to a gene encoding a zinc-finger protein on chromosome 2p15 [J].

Menzel, Stephan ;

Garner, Chad ;

Gut, Ivo ;

Matsuda, Fumihiko ;

Yamaguchi, Masao ;

Heath, Simon ;

Foglio, Mario ;

Zelenika, Diana ;

Boland, Anne ;

Rooks, Helen ;

Best, Steve ;

Spector, Tim D. ;

Farrall, Martin ;

Lathrop, Mark ;

Thein, Swee Lay .

NATURE GENETICS, 2007, 39 (10) :1197-1199

[9] Emerging Genetic Therapy for Sickle Cell Disease [J].

Orkin, Stuart H. ;

Bauer, Daniel E. .

ANNUAL REVIEW OF MEDICINE, VOL 70, 2019, 70 :257-271

[10] HYDROXYUREA ENHANCES FETAL HEMOGLOBIN PRODUCTION IN SICKLE-CELL-ANEMIA [J].

PLATT, OS ;

ORKIN, SH ;

DOVER, G ;

BEARDSLEY, GP ;

MILLER, B ;

NATHAN, DG .

JOURNAL OF CLINICAL INVESTIGATION, 1984, 74 (02) :652-656

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