Zobrazeno 1 - 7
of 7
pro vyhledávání: '"Kaitly J. Woodard"'
Autor:
Kaitly J. Woodard, Phillip A. Doerfler, Kalin D. Mayberry, Akshay Sharma, Rachel Levine, Jonathan Yen, Virginia Valentine, Lance E. Palmer, Marc Valentine, Mitchell J. Weiss
Publikováno v:
Disease Models & Mechanisms, Vol 15, Iss 6 (2022)
We characterized the human β-like globin transgenes in two mouse models of sickle cell disease (SCD) and tested a genome-editing strategy to induce red blood cell fetal hemoglobin (HbF; α2γ2). Berkeley SCD mice contain four to 22 randomly arranged
Externí odkaz:
https://doaj.org/article/c7a2b29fe3814d94a95e8c2dce8aa30e
Autor:
Jonathan Yen, Gregory A. Newby, Kalin Mayberry, Akshay Sharma, Michelle Richter, Thiyagaraj Mayuranathan, Kevin T. Zhao, Cicera R. Lazzarotto, Christophe Lechauve, Theodosia A. Kalfa, Elizabeth Thaman, Luke W. Koblan, Shondra M. Pruett-Miller, Heather Sheppard-Tillman, David R. Liu, Mitchell J. Weiss, Shengdar Q. Tsai, John F. Tisdale, Kaitly J. Woodard, Yoonjeong Jang, Christopher J. Podracky, Kelcee A. Everette, Shannon M. Miller, Tina Wang, Shaina N. Porter, Anton P. McCaffrey, Jordana M. Henderson, Yichao Li, Yu Yao
Publikováno v:
Nature
Sickle cell disease (SCD) is caused by a mutation in the β-globin gene HBB1. We used a custom adenine base editor (ABE8e-NRCH)2,3 to convert the SCD allele (HBBS) into Makassar β-globin (HBBG), a non-pathogenic variant4,5. Ex vivo delivery of mRNA
Autor:
Jing Zeng, Byoung Y. Ryu, Kaitly J. Woodard, Stephanie Fowler, John F. Tisdale, Shengdar Q. Tsai, Jean-Yves Metais, Shondra M. Pruett-Miller, Cicera R. Lazzarotto, Yu Yao, Kevin Luk, Yuxuan Wu, Sagar Keriwala, Michael D. Neel, Daniel E. Bauer, Samuel T. Peters, S. Scott Perry, Scot A. Wolfe, Shaina N. Porter, Mitchell J. Weiss, Thiyagaraj Mayuranathan, Varun Katta, Naoya Uchida, Akshay Sharma, Matthew M. Hsieh, Devlin Shea, Phillip A. Doerfler
Publikováno v:
Blood Advances. 3:3379-3392
Induction of fetal hemoglobin (HbF) via clustered regularly interspaced short palindromic repeats/Cas9–mediated disruption of DNA regulatory elements that repress γ-globin gene (HBG1 and HBG2) expression is a promising therapeutic strategy for sic
Autor:
Jingjing Zhang, Shaina N. Porter, Jonathan Yen, Kalin Mayberry, Kaitly J. Woodard, Gregory A. Newby, Jonathan S Yen, Kelcee A. Everette, Mitchell J. Weiss, Anton P. McCaffrey, Thiyagaraj Mayuranathan, Jordana M. Henderson, John F. Tisdale, Shengdar Q. Tsai, Yu Yao, David R. Liu
Publikováno v:
Blood. 136:13-14
Sickle cell disease (SCD) is a chronic, life-altering multisystem disorder that affects millions of individuals worldwide. Strategies for genetic therapy of autologous SCD hematopoietic stem cells (HSCs) include lentiviral vector (LV) delivery of an
Adenosine Base Editing of γ-Globin Promoters Induces Fetal Hemoglobin and Inhibit Erythroid Sickling
Autor:
John F. Tisdale, Jingjing Zhang, Shengdar Q. Tsai, Yu Yao, David R. Liu, Gregory A. Newby, Akshay Sharma, Kaitly J. Woodard, Thiyagaraj Mayuranathan, Mitchell J. Weiss, Shondra M. preutt-Miller, Jonathan Yen, Shaina N. Porter, Kalin Mayberry
Publikováno v:
Blood. 136:21-22
Rare variants in the γ-globin (HBG2 and HBG1) promoters cause sustained postnatal expression of fetal hemoglobin (HbF, α2γ2) in red blood cells (RBCs). This benign condition is termed hereditary persistence of fetal hemoglobin (HPFH). Individuals
Autor:
Thiyagaraj Mayuranathan, Sagar Keriwala, Varun Katta, Kaitly J. Woodard, Stephanie Fowler, Mitchell J. Weiss, Jing Zeng, Yuxuan Wu, Yu Yao, S. Scott Perry, Kevin Luk, Samuel T. Peters, John F. Tisdale, Michael D. Neel, Cicera R. Lazzarotto, Byoung Y. Ryu, Jean-Yves Metais, Shengdar Q. Tsai, Naoya Uchida, Phillip A. Doerfler, Daniel E. Bauer, Matthew M. Hsieh, Devlin Shea, Akshay Sharma, Scot A. Wolfe, Shaina N. Porter, Shondra M. Pruett-Miller
Publikováno v:
Blood. 134:2066-2066
Induction of fetal hemoglobin (HbF, α2γ2) via genome editing-mediated disruption of DNA regulatory elements that repress expression of γ-globin genes (HBG1 and HBG2) is a promising therapeutic strategy for b-hemoglobinopathies including sickle cel
Autor:
Elizabeth A. Traxler, Yu Yao, Gerd A. Blobel, Yukio Nakamura, Chunliang Li, Mitchell J. Weiss, Ryo Kurita, Yong Dong Wang, Jim R. Hughes, Kaitly J. Woodard, Ross C. Hardison
Publikováno v:
Nature medicine. 22(9)
Disorders resulting from mutations in the hemoglobin subunit beta gene (HBB; which encodes β-globin), mainly sickle cell disease (SCD) and β-thalassemia, become symptomatic postnatally as fetal γ-globin expression from two paralogous genes, hemogl