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1
Highly efficient therapeutic gene editing of human hematopoietic stem cells
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Artigo
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Highly efficient therapeutic gene editing of human hematopoietic stem cells

Wu, Yuxuan ; Zeng, Jing ; Roscoe, Benjamin P ; Liu, Pengpeng ; Yao, Qiuming ; Lazzarotto, Cicera R ; Clement, Kendell ; Cole, Mitchel A ; Luk, Kevin ; Baricordi, Cristina ; Shen, Anne H ; Ren, Chunyan ; Esrick, Erica B ; Manis, John P ; Dorfman, David M ; Williams, David A ; Biffi, Alessandra ; Brugnara, Carlo ; Biasco, Luca ; Brendel, Christian ; Pinello, Luca ; Tsai, Shengdar Q ; Wolfe, Scot A ; Bauer, Daniel E

Nature medicine, 2019-05, Vol.25 (5), p.776-783 [Periódico revisado por pares]

United States: Nature Publishing Group

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2
Global epidemiology of haemoglobin disorders and derived service indicators
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Artigo
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Global epidemiology of haemoglobin disorders and derived service indicators

MODELL, Bernadette ; DARLISON, Matthew

Bulletin of the World Health Organization, 2008-06, Vol.86 (6), p.480-487 [Periódico revisado por pares]

Genève: Organisation mondiale de la santé

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3
A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells
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Artigo
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A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

Vakulskas, Christopher A ; Dever, Daniel P ; Rettig, Garrett R ; Turk, Rolf ; Jacobi, Ashley M ; Collingwood, Michael A ; Bode, Nicole M ; McNeill, Matthew S ; Yan, Shuqi ; Camarena, Joab ; Lee, Ciaran M ; Park, So Hyun ; Wiebking, Volker ; Bak, Rasmus O ; Gomez-Ospina, Natalia ; Pavel-Dinu, Mara ; Sun, Wenchao ; Bao, Gang ; Porteus, Matthew H ; Behlke, Mark A

Nature medicine, 2018-08, Vol.24 (8), p.1216-1224 [Periódico revisado por pares]

United States: Nature Publishing Group

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4
Extracellular hemin crisis triggers acute chest syndrome in sickle mice
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Artigo
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Extracellular hemin crisis triggers acute chest syndrome in sickle mice

Ghosh, Samit ; Adisa, Olufolake Adetoro ; Chappa, Prasanthi ; Tan, Fang ; Jackson, Kesmic Ann ; Archer, David Robert ; Ofori-Acquah, Solomon Fiifi

The Journal of clinical investigation, 2013-11, Vol.123 (11), p.4809-4820 [Periódico revisado por pares]

United States: American Society for Clinical Investigation

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5
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
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Artigo
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CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

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 (London), 2016-11, Vol.539 (7629), p.384-389 [Periódico revisado por pares]

England: Springer Science and Business Media LLC

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6
Intravascular hemolysis and the pathophysiology of sickle cell disease
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Artigo
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Intravascular hemolysis and the pathophysiology of sickle cell disease

Kato, Gregory J ; Steinberg, Martin H ; Gladwin, Mark T

The Journal of clinical investigation, 2017-03, Vol.127 (3), p.750-760 [Periódico revisado por pares]

United States: American Society for Clinical Investigation

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7
Therapeutic base editing of human hematopoietic stem cells
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Artigo
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Therapeutic base editing of human hematopoietic stem cells

Zeng, Jing ; Wu, Yuxuan ; Ren, Chunyan ; Bonanno, Jasmine ; Shen, Anne H ; Shea, Devlin ; Gehrke, Jason M ; Clement, Kendell ; Luk, Kevin ; Yao, Qiuming ; Kim, Rachel ; Wolfe, Scot A ; Manis, John P ; Pinello, Luca ; Joung, J Keith ; Bauer, Daniel E

Nature medicine, 2020-04, Vol.26 (4), p.535-541 [Periódico revisado por pares]

United States: Nature Publishing Group

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8
Neutrophil ageing is regulated by the microbiome
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Artigo
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Neutrophil ageing is regulated by the microbiome

Zhang, Dachuan ; Chen, Grace ; Manwani, Deepa ; Mortha, Arthur ; Xu, Chunliang ; Faith, Jeremiah J ; Burk, Robert D ; Kunisaki, Yuya ; Jang, Jung-Eun ; Scheiermann, Christoph ; Merad, Miriam ; Frenette, Paul S

Nature (London), 2015-09, Vol.525 (7570), p.528-532 [Periódico revisado por pares]

England

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9
Treating sickle cell anemia
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Artigo
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Treating sickle cell anemia

Tisdale, John F ; Thein, Swee Lay ; Eaton, William A

Science (American Association for the Advancement of Science), 2020-03, Vol.367 (6483), p.1198-1199 [Periódico revisado por pares]

United States: The American Association for the Advancement of Science

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10
Prevalence of sickle cell trait and needs assessment for uptake of sickle cell screening among secondary school students in Kampala City, Uganda
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Artigo
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Prevalence of sickle cell trait and needs assessment for uptake of sickle cell screening among secondary school students in Kampala City, Uganda

Namukasa, Shamim ; Maina, Rose ; Nakaziba, Sarah ; Among, Grace ; Asasira, Lydia ; Mayambala, Posiano ; Atukwatse, Joseph ; Namuguzi, Mary ; Sarki, Ahmed M Maric, Gorica

PloS one, 2024-01, Vol.19 (1), p.e0296119-e0296119 [Periódico revisado por pares]

United States: Public Library of Science

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