|
|
|
HBB-deficient Macaca fascicularis monkey presents with human β-thalassemia |
Yan Huang1, Chenhui Ding2, Puping Liang2,3, Duanduan Li1, Yu Tang1, Wei Meng1, Hongwei Sun3, Hongyu Lu1, Yu Chen3, Xueying Chen1, Qunshan Huang1, Jianpei Fang4, Canquan Zhou2( ), Shihua Yang1(), Junjiu Huang2,3() |
1. College of Veterinary Medicine Key Laboratory of Comprehensive Prevention and Control for Severe Clinical Animal Diseases of Guangdong Province, South China Agricultural University, Guangzhou 510642, China
2. Key Laboratory of Reproductive Medicine of Guangdong Province, The First Affiliated Hospital and School of Life Sciences, Sun Yatsen University, Guangzhou 510275, China
3. MOE Key Laboratory of Gene Function and Regulation, State Key Laboratory of Biocontrol, School of Life Sciences, SunYat-sen University, Guangzhou 510275, China
4. Department of Pediatrics, Second Affiliated Hospital, Sun Yat-sen University, Guangzhou 510120, China |
|
|
|
|
|
|
Corresponding Author(s):
Canquan Zhou,Shihua Yang,Junjiu Huang
|
|
Issue Date: 25 July 2019
|
|
| 1 |
MA El-Brolosy, Z Kontarakis, A Rossi, C Kuenne, S Günther, N Fukuda, K Kikhi, GLM Boezio, CM Takacs, SL Laiet al. (2019) Genetic compensation triggered by mutant mRNA degradation. Nature 568:193–197
https://doi.org/10.1038/s41586-019-1064-z
|
| 2 |
MM Eldibany, KF Totonchi, NJ Joseph, D Rhone (1999) Usefulness of certain red blood cell indices in diagnosing and differentiating thalassemia trait from iron-deficiency anemia. Am J Clin Pathol 111:676–682
https://doi.org/10.1093/ajcp/111.5.676
|
| 3 |
A Glaser, B McColl, J Vadolas (2015) The therapeutic potential of genome editing for beta-thalassemia. F1000Research.
https://doi.org/10.12688/f1000research.7087.1
|
| 4 |
Y Huo, SC McConnell, TM Ryan (2009) Preclinical transfusiondependent humanized mouse model of beta thalassemia major. Blood 113:4763–4770
https://doi.org/10.1182/blood-2008-12-197012
|
| 5 |
Z Liu, Y Cai, Y Wang, Y Nie, C Zhang, Y Xu, X Zhang, Y Lu, Z Wang, M Pooet al. (2018) Cloning of macaque monkeys by somatic cell nuclear transfer. Cell 172:881–887, e887
https://doi.org/10.1016/j.cell.2018.01.020
|
| 6 |
Z Ma, P Zhu, H Shi, L Guo, Q Zhang, Y Chen, S Chen, Z Zhang, J Peng, J Chen (2019) PTC-bearing mRNA elicits a genetic compensation response via Upf3a and COMPASS components. Nature 568:259–263
https://doi.org/10.1038/s41586-019-1057-y
|
| 7 |
B McColl, J Vadolas (2016) Animal models of beta-hemoglobinopathies: utility and limitations. J Blood Med 7:263–274
https://doi.org/10.2147/JBM.S87955
|
| 8 |
AW Nienhuis, DG Nathan (2012) Pathophysiology and clinical manifestations of the beta-thalassemias. Cold Spring Harb Perspect Med 2:a011726
https://doi.org/10.1101/cshperspect.a011726
|
| 9 |
R Origa (2017) beta-Thalassemia. Genet Med Off J Am Coll Med Genet 19:609–619
https://doi.org/10.1038/gim.2016.173
|
| 10 |
KA Phillips, KL Bales, JP Capitanio, A Conley, PW Czoty, BA ’t Hart, WD Hopkins, SL Hu, LA Miller, MA Naderet al. (2014) Why primate models matter. Am J Primatol 76:801–827
https://doi.org/10.1002/ajp.22281
|
| 11 |
R Scott, S Anyaibe, A Yancey, V Headings (1986) Fetal hemoglobin distributions among adult baboon and macaque species. Primates 27:259–268
https://doi.org/10.1007/BF02382604
|
| 12 |
CC So, AC So, AY Chan, ST Tsang, ES Ma, LC Chan (2009) Detection and characterisation of beta-globin gene cluster deletions in Chinese using multiplex ligation-dependent probe amplification. J Clin Pathol 62:1107–1111
https://doi.org/10.1136/jcp.2009.067538
|
| 13 |
AA Thompson, MC Walters, J Kwiatkowski, JEJ Rasko, JA Ribeil, S Hongeng, E Magrin, GJ Schiller, E Payen, M Semeraroet al. (2018) Gene therapy in patients with transfusion-dependent betathalassemia. N Engl J Med 378:1479–1493
https://doi.org/10.1056/NEJMoa1705342
|
| 14 |
F Xiong, M Sun, X Zhang, R Cai, Y Zhou, J Lou, L Zeng, Q Sun, Q Xiao, X Shanget al. (2010) Molecular epidemiological survey of haemoglobinopathies in the Guangxi Zhuang Autonomous Region of Southern China. Clin Genet 78:139–148
https://doi.org/10.1111/j.1399-0004.2010.01430.x
|
| 15 |
W Zhang, H Wan, G Feng, J Qu, J Wang, Y Jing, R Ren, Z Liu, L Zhang, Z Chenet al. (2018) SIRT6 deficiency results in developmental retardation in cynomolgus monkeys. Nature 560:661–665
https://doi.org/10.1038/s41586-018-0437-z
|
|
Viewed |
|
|
|
Full text
|
|
|
|
|
Abstract
|
|
|
|
|
Cited |
|
|
|
|
| |
Shared |
|
|
|
|
| |
Discussed |
|
|
|
|
