Resultats de la cerca - He, Zhenning

A human embryonic hemoglobin inhibits Hb S polymerization in vitro and restores a normal phenotype to mouse models of sickle cell disease per He, Zhenning, Russell, J. Eric

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Dynamic posttranscriptional regulation of ϵ-globin gene expression in vivo per He, Zhenning, Russell, J. Eric

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Structural determinants of human ζ-globin mRNA stability per He, Zhenning, Song, Decheng, van Zalen, Sebastiaan, Russell, J Eric

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Structure of fully liganded Hb ζ(2)β(2) (s) trapped in a tense conformation per Safo, Martin K., Ko, Tzu-Ping, Abdulmalik, Osheiza, He, Zhenning, Wang, Andrew H.-J., Schreiter, Eric R., Russell, J. Eric

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AAV Gene Therapy Corrects OTC Deficiency and Prevents Liver Fibrosis in Aged OTC-Knock Out Heterozygous Mice per Wang, Lili, Bell, Peter, Morizono, Hiroki, He, Zhenning, Pumbo, Elena, Yu, Hongwei, White, John, Batshaw, Mark L., Wilson, James M.

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Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia per Wang, Lili, Muthuramu, Ilayaraja, Somanathan, Suryanarayan, Zhang, Hong, Bell, Peter, He, Zhenning, Yu, Hongwei, Zhu, Yanqing, Tretiakova, Anna P., Wilson, James M.

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Effects of Self-Complementarity, Codon Optimization, Transgene, and Dose on Liver Transduction with AAV8 per Bell, Peter, Wang, Lili, Chen, Shu-Jen, Yu, Hongwei, Zhu, Yanqing, Nayal, Mohamad, He, Zhenning, White, John, Lebel-Hagan, Deborah, Wilson, James M.

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A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice per Yang, Yang, Wang, Lili, Bell, Peter, McMenamin, Deirdre, He, Zhenning, White, John, Yu, Hongwei, Xu, Chenyu, Morizono, Hiroki, Musunuru, Kiran, Batshaw, Mark L., Wilson, James M.

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Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9 per Wang, Lili, Breton, Camilo, Warzecha, Claude C., Bell, Peter, Yan, Hanying, He, Zhenning, White, John, Zhu, Yanqing, Li, Mingyao, Buza, Elizabeth L., Jantz, Derek, Wilson, James M.

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A mutation-independent CRISPR-Cas9–mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency per Wang, Lili, Yang, Yang, Breton, Camilo, Bell, Peter, Li, Mingyao, Zhang, Jia, Che, Yan, Saveliev, Alexei, He, Zhenning, White, John, Latshaw, Caitlin, Xu, Chenyu, McMenamin, Deirdre, Yu, Hongwei, Morizono, Hiroki, Batshaw, Mark L., Wilson, James M.

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Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression per Wang, Lili, Warzecha, Claude C., Kistner, Alexander, Chichester, Jessica A., Bell, Peter, Buza, Elizabeth L., He, Zhenning, Pampena, M. Betina, Couthouis, Julien, Sethi, Sunjay, McKeever, Kathleen, Betts, Michael R., Kakkis, Emil, Wilson, James M., Wadsworth, Samuel, Sullivan, Barbara A.

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