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Autor:
Helen Segal, Sruthi Mantri, M. Kyle Cromer, Neehar Bhatia, Maria Grazia Roncarolo, Annalisa Lattanzi, Ciaran M. Lee, David DiGiusto, Rasmus O. Bak, Carsten T. Charlesworth, Josefin Kenrick, Jason Skowronski, Matthew H. Porteus, J. Fraser Wright, Richard L. Frock, Daniel P. Dever, Narae Talbott, Christopher A. Vakulskas, Joab Camarena, Gang Bao, Waracharee Srifa, Premanjali Lahiri, John F. Tisdale
Publikováno v:
Sci Transl Med
Lattanzi, A, Camarena, J, Lahiri, P, Segal, H, Srifa, W, Vakulskas, C A, Frock, R L, Kenrick, J, Lee, C, Talbott, N, Skowronski, J, Cromer, M K, Charlesworth, C T, Bak, R O, Mantri, S, Bao, G, DiGiusto, D, Tisdale, J, Wright, J F, Bhatia, N, Roncarolo, M G, Dever, D P & Porteus, M H 2021, ' Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease ', Science Translational Medicine, vol. 13, no. 598, eabf2444 . https://doi.org/10.1126/scitranslmed.abf2444
Lattanzi, A, Camarena, J, Lahiri, P, Segal, H, Srifa, W, Vakulskas, C A, Frock, R L, Kenrick, J, Lee, C, Talbott, N, Skowronski, J, Cromer, M K, Charlesworth, C T, Bak, R O, Mantri, S, Bao, G, DiGiusto, D, Tisdale, J, Wright, J F, Bhatia, N, Roncarolo, M G, Dever, D P & Porteus, M H 2021, ' Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease ', Science Translational Medicine, vol. 13, no. 598, eabf2444 . https://doi.org/10.1126/scitranslmed.abf2444
Sickle cell disease (SCD) is the most common monogenic serious disease with 300,000 births annually worldwide. SCD is autosomal recessive from a single point mutation in codon six of the β-globin gene (HBB) resulting in sickle hemoglobin. Ex vivo β