从幼犬到成年犬：Ia型糖原贮积病犬模型中肝细胞的编辑 - Suppr

From Puppies to adults: editing of hepatocytes in a canine model of glycogen storage disease type Ia.

作者信息

Chandler Randy J

机构信息

Metabolic Medicine Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.

出版信息

Mol Ther Methods Clin Dev. 2023 May 9;29:347-349. doi: 10.1016/j.omtm.2023.04.006. eCollection 2023 Jun 8.

DOI:10.1016/j.omtm.2023.04.006

PMID:37206367

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10188620/

Abstract

摘要

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/23bf/10188620/fdf83441a276/gr1.jpg

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From Puppies to adults: editing of hepatocytes in a canine model of glycogen storage disease type Ia.从幼犬到成年犬：Ia型糖原贮积病犬模型中肝细胞的编辑

Mol Ther Methods Clin Dev. 2023 May 9;29:347-349. doi: 10.1016/j.omtm.2023.04.006. eCollection 2023 Jun 8.

Canine model and genomic structural organization of glycogen storage disease type Ia (GSD Ia).犬糖原贮积病Ia型（GSD Ia）模型及基因组结构组织

Vet Pathol. 2001 Jan;38(1):83-91. doi: 10.1354/vp.38-1-83.

Isolation and nucleotide sequence of canine glucose-6-phosphatase mRNA: identification of mutation in puppies with glycogen storage disease type Ia.犬葡萄糖-6-磷酸酶mRNA的分离与核苷酸序列：Ia型糖原贮积病幼犬中突变的鉴定。

Biochem Mol Med. 1997 Aug;61(2):168-77. doi: 10.1006/bmme.1997.2600.

Glycogen storage disease type Ia in two littermate Maltese puppies.两只同窝马耳他幼犬患Ia型糖原贮积病。

Vet Pathol. 1995 Sep;32(5):460-5. doi: 10.1177/030098589503200502.

Hepatocytes contribute to residual glucose production in a mouse model for glycogen storage disease type Ia.肝细胞在糖原贮积病 Ia 型的小鼠模型中有助于残余葡萄糖的产生。

Hepatology. 2017 Dec;66(6):2042-2054. doi: 10.1002/hep.29389. Epub 2017 Oct 30.

Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors.在犬类糖原贮积病Ia型模型中，使用腺相关病毒（AAV）载体递送葡萄糖-6-磷酸酶。

Gene Ther. 2002 Aug;9(15):1015-22. doi: 10.1038/sj.gt.3301728.

Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia.苯扎贝特增强腺相关病毒载体介导的Ⅰ型糖原贮积病的基因组编辑。

Mol Ther Methods Clin Dev. 2019 Feb 10;13:265-273. doi: 10.1016/j.omtm.2019.02.002. eCollection 2019 Jun 14.

Correction of metabolic abnormalities in a mouse model of glycogen storage disease type Ia by CRISPR/Cas9-based gene editing.通过基于 CRISPR/Cas9 的基因编辑纠正糖原贮积病 Ia 型小鼠模型中的代谢异常。

Mol Ther. 2021 Apr 7;29(4):1602-1610. doi: 10.1016/j.ymthe.2020.12.027. Epub 2020 Dec 23.

Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease.糖原贮积病小鼠和犬模型基因治疗后肝肿瘤的发病机制

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Stone forming risk factors in patients with type Ia glycogen storage disease.Ⅰa 型糖原贮积病患者结石形成的风险因素。

J Urol. 2010 Mar;183(3):1022-5. doi: 10.1016/j.juro.2009.11.040. Epub 2010 Jan 21.

本文引用的文献

Genome editing using Cas9 in a canine model of glycogen storage disease Ia.在糖原贮积病Ia型犬模型中使用Cas9进行基因组编辑。

Mol Ther Methods Clin Dev. 2023 Mar 9;29:108-119. doi: 10.1016/j.omtm.2023.03.001. eCollection 2023 Jun 8.

Genome editing technologies to treat rare liver diseases.用于治疗罕见肝脏疾病的基因组编辑技术。

Transl Gastroenterol Hepatol. 2020 Apr 5;5:23. doi: 10.21037/tgh.2019.10.10. eCollection 2020.

Gene therapy for glycogen storage diseases.糖原贮积病的基因治疗。

Hum Mol Genet. 2019 Oct 1;28(R1):R31-R41. doi: 10.1093/hmg/ddz133.

Bezafibrate induces autophagy and improves hepatic lipid metabolism in glycogen storage disease type Ia.苯扎贝特诱导自噬并改善糖原贮积病 Ia 型的肝脂代谢。

Hum Mol Genet. 2019 Jan 1;28(1):143-154. doi: 10.1093/hmg/ddy343.

In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.体内锌指核酸酶介导的葡萄糖-6-磷酸酶转基因靶向整合促进ⅠA型糖原贮积病小鼠存活。

Mol Ther. 2016 Apr;24(4):697-706. doi: 10.1038/mt.2016.35. Epub 2016 Feb 11.

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.载体设计影响腺相关病毒基因治疗后的肝脏遗传毒性。

J Clin Invest. 2015 Feb;125(2):870-80. doi: 10.1172/JCI79213. Epub 2015 Jan 20.

Gene targeting to the ROSA26 locus directed by engineered zinc finger nucleases.通过工程化锌指核酸酶靶向 ROSA26 基因座的基因打靶。

Nucleic Acids Res. 2012 Apr;40(8):3741-52. doi: 10.1093/nar/gkr1214. Epub 2011 Dec 14.

Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo.重组腺病毒载体基因组在体内细胞周期中的游离型持续存在。

J Virol. 2003 Jul;77(13):7689-95. doi: 10.1128/jvi.77.13.7689-7695.2003.

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From Puppies to adults: editing of hepatocytes in a canine model of glycogen storage disease type Ia.

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