CRISPR-mediated gene editing to rescue haploinsufficient obesity syndrome
Published date: 15 Oct 2019
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Zhifeng Wang , Liu Yang , Shen Qu , Chao Zhang . CRISPR-mediated gene editing to rescue haploinsufficient obesity syndrome[J]. Protein & Cell, 2019 , 10(10) : 705 -708 . DOI: 10.1007/s13238-019-0635-y
1 |
Akcakaya P, Bobbin ML, Guo JA, Malagon-Lopez J, Clement K, Garcia SP, Fellows MD, Porritt MJ, Firth MA, Carreras A
|
2 |
Amoasii L, Hildyard JCW, Li H, Sanchez-Ortiz E, Mireault A, Caballero D, Harron R, Stathopoulou TR, Massey C, Shelton JM
|
3 |
Argyropoulos G, Brown AM, Willi SM, Zhu J, He Y, Reitman M, Gevao SM, Spruill I, Garvey WT (1998) Effects of mutations in the human uncoupling protein 3 gene on the respiratory quotient and fat oxidation in severe obesity and type 2 diabetes. J Clin Investig 102:1345–1351
|
4 |
Asai M, Ramachandrappa S, Joachim M, Shen Y, Zhang R, Nuthalapati N, Ramanathan V, Strochlic DE, Ferket P, Linhart K
|
5 |
Bakondi B, Lv W, Lu B, Jones MK, Tsai Y, Kim KJ, Levy R, Akhtar AA, Breunig JJ, Svendsen CN
|
6 |
Balthasar N, Dalgaard LT, Lee CE, Yu J, Funahashi H, Williams T, Ferreira M, Tang V, McGovern RA, Kenny CD
|
7 |
Boettcher M, McManus MT (2015) Choosing the right tool for the job: RNAi, TALEN, or CRISPR. Mol Cell 58:575–585
|
8 |
Chapdelaine P, Gerard C, Sanchez N, Cherif K, Rousseau J, Ouellet DL, Jauvin D, Tremblay JP (2016) Development of an AAV9 coding for a 3XFLAG-TALEfrat#8-VP64 able to increase in vivo the human frataxin in YG8R mice. Gene Ther 23:606–614
|
9 |
Church C, Moir L, McMurray F, Girard C, Banks GT, Teboul L, Wells S, Bruning JC, Nolan PM, Ashcroft FM
|
10 |
Cohen J (2018) In dogs, CRISPR fixes a muscular dystrophy. Science 361:835
|
11 |
Creemers JW, Choquet H, Stijnen P, Vatin V, Pigeyre M, Beckers S, Meulemans S, Than ME, Yengo L, Tauber M
|
12 |
Ding Q, Strong A, Patel KM, Ng SL, Gosis BS, Regan SN, Cowan CA, Rader DJ, Musunuru K (2014) Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing. Circ Res 115:488–492
|
13 |
Farooqi IS, Keogh JM, Yeo GS, Lank EJ, Cheetham T, O’Rahilly S (2003) Clinical spectrum of obesity and mutations in the melanocortin 4 receptor gene. N Engl J Med 348:1085–1095
|
14 |
Hohenstein P, Slight J, Ozdemir DD, Burn SF, Berry R, Hastie ND (2008) High-efficiency Rosa26 knock-in vector construction for Cre-regulated overexpression and RNAi. Pathogenetics 1:3
|
15 |
Huszar D, Lynch CA, Fairchild-Huntress V, Dunmore JH, Fang Q, Berkemeier LR, Gu W, Kesterson RA, Boston BA, Cone RD
|
16 |
Kannisto K, Pietilainen KH, Ehrenborg E, Rissanen A, Kaprio J, Hamsten A, Yki-Jarvinen H (2004) Overexpression of 11beta-hydroxysteroid dehydrogenase-1 in adipose tissue is associated with acquired obesity and features of insulin resistance: studies in young adult monozygotic twins. J Clin Endocrinol Metab 89:4414–4421
|
17 |
Kublaoui BM, Holder JL Jr, Tolson KP, Gemelli T, Zinn AR (2006) SIM1 overexpression partially rescues agouti yellow and diet- induced obesity by normalizing food intake. Endocrinology 147:4542–4549
|
18 |
Liang P, Xu Y, Zhang X, Ding C, Huang R, Zhang Z, Lv J, Xie X, Chen Y, Li Y
|
19 |
Liu C, Zhang L, Liu H, Cheng K (2017) Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications. J Control Release 266:17–26
|
20 |
Long C, Amoasii L, Mireault AA, McAnally JR, Li H, Sanchez-Ortiz E, Bhattacharyya S, Shelton JM, Bassel-Duby R, Olson EN (2016) Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science 351:400–403
|
21 |
Ma Y, Zhang L, Huang X (2014) Genome modification by CRISPR/Cas9. FEBS J 281:5186–5193
|
22 |
Matharu N, Rattanasopha S, Tamura S, Maliskova L, Wang Y, Bernard A, Hardin A, Eckalbar WL, Vaisse C, Ahituv N (2019) CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency. Science 363:eaau0629
|
23 |
Michaud JL, Boucher F, Melnyk A, Gauthier F, Goshu E, Levy E, Mitchell GA, Himms-Hagen J, Fan CM (2001) Sim1 haploinsuf- ficiency causes hyperphagia, obesity and reduction of the paraventricular nucleus of the hypothalamus. Hum Mol Genet 10:1465–1473
|
24 |
Nelson CE, Hakim CH, Ousterout DG, Thakore PI, Moreb EA, Castellanos Rivera RM, Madhavan S, Pan X, Ran FA, Yan WX
|
25 |
Pignani S, Zappaterra F, Barbon E, Follenzi A, Bovolenta M, Bernardi F, Branchini A, Pinotti M (2019) Tailoring the CRISPR system to transactivate coagulation gene promoters in normal and mutated contexts. Biochim Biophys Acta Gene Regul Mech. https://doi.org/10.1016/j.bbagrm.2019.04.002
|
26 |
Rankinen T, Zuberi A, Chagnon YC, Weisnagel SJ, Argyropoulos G, Walts B, Perusse L, Bouchard C (2006) The human obesity gene map: the 2005 update. Obesity 14:529–644
|
27 |
Rossidis AC, Stratigis JD, Chadwick AC, Hartman HA, Ahn NJ, Li H, Singh K, Coons BE, Li L, Lv W
|
28 |
Savell KE, Bach SV, Zipperly ME, Revanna JS, Goska NA, Tuscher JJ, Duke CG, Sultan FA, Burke JN, Williams D
|
29 |
Seeger C, Sohn JA (2014) Targeting hepatitis B virus with CRISPR/Cas9. Mol Ther Nucleic Acids 3:e216
|
30 |
Sera T (2009) Zinc-finger-based artificial transcription factors and their applications. Adv Drug Deliv Rev 61:513–526
|
31 |
Shah BP, Vong L, Olson DP, Koda S, Krashes MJ, Ye C, Yang Z, Fuller PM, Elmquist JK, Lowell BB (2014) MC4R-expressing glutamatergic neurons in the paraventricular hypothalamus regulate feeding and are synaptically connected to the parabrachial nucleus. Proc Natl Acad Sci USA 111:13193–13198
|
32 |
Shinohara ET, Kaminski JM, Segal DJ, Pelczar P, Kolhe R, Ryan T, Coates CJ, Fraser MJ, Handler AM, Yanagimachi R
|
33 |
Soriano V (2017) Hot news: gene therapy with CRISPR/Cas9 coming to age for HIV cure. AIDS Rev 19:167–172
|
34 |
Tabebordbar M, Zhu K, Cheng JKW, Chew WL, Widrick JJ, Yan WX, Maesner C, Wu EY, Xiao R, Ran FA
|
35 |
Vaisse C, Clement K, Durand E, Hercberg S, Guy-Grand B, Froguel P (2000) Melanocortin-4 receptor mutations are a frequent and heterogeneous cause of morbid obesity. J Clin Investig 106:253–262
|
36 |
Wang L, Smith J, Breton C, Clark P, Zhang J, Ying L, Che Y, Lape J, Bell P, Calcedo R
|
37 |
Wang X, Raghavan A, Chen T, Qiao L, Zhang Y, Ding Q, Musunuru K (2016) CRISPR-Cas9 targeting of PCSK9 in human hepatocytes in vivo-brief report. Arterioscler Thromb Vasc Biol 36:783–786
|
38 |
Xiong L, Wu F, Wu Q, Xu L, Cheung OK, Kang W, Mok MT, Szeto LLM, Lun CY, Lung RW
|
39 |
Xu Y, Wu Z, Sun H, Zhu Y, Kim ER, Lowell BB, Arenkiel BR, Xu Y, Tong Q (2013) Glutamate mediates the function of melanocortin receptor 4 on Sim1 neurons in body weight regulation. Cell Metab 18:860–870
|
40 |
Zou Y, Lu P, Shi J, Liu W, Yang M, Zhao S, Chen N, Chen M, Sun Y, Gao A
|
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