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囊性纤维化跨膜传导调节因子-ΔF508兔的培育

Production of CFTR-ΔF508 Rabbits.

作者信息

Yang Dongshan, Liang Xiubin, Pallas Brooke, Hoenerhoff Mark, Ren Zhuoying, Han Renzhi, Zhang Jifeng, Chen Y Eugene, Jin Jian-Ping, Sun Fei, Xu Jie

机构信息

Center for Advanced Models for Translational Sciences and Therapeutics, University of Michigan Medical Center, University of Michigan Medical School, Ann Arbor, MI, United States.

Unit for Laboratory Animal Medicine, University of Michigan Medical School, Ann Arbor, MI, United States.

出版信息

Front Genet. 2021 Jan 22;11:627666. doi: 10.3389/fgene.2020.627666. eCollection 2020.

Abstract

Cystic Fibrosis (CF) is a lethal autosomal recessive disease caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR). The most common mutation is the deletion of phenylalanine residue at position 508 (ΔF508). Here we report the production of CFTR-ΔF508 rabbits by CRISPR/Cas9-mediated gene editing. After microinjection and embryo transfer, 77 kits were born, of which five carried the ΔF508 mutation. To confirm the germline transmission, one male ΔF508 founder was bred with two wild-type females and produced 16 F1 generation kits, of which six are heterozygous ΔF508/WT animals. Our work adds CFTR-ΔF508 rabbits to the toolbox of CF animal models for biomedical research.

摘要

囊性纤维化(CF)是一种致命的常染色体隐性疾病,由编码囊性纤维化跨膜传导调节因子(CFTR)的基因突变引起。最常见的突变是第508位苯丙氨酸残基缺失(ΔF508)。在此,我们报告通过CRISPR/Cas9介导的基因编辑产生了CFTR-ΔF508兔。显微注射和胚胎移植后,出生了77只幼兔,其中5只携带ΔF508突变。为了确认种系传递,一只雄性ΔF508奠基兔与两只野生型雌性兔交配,产生了16只F1代幼兔,其中6只是杂合ΔF508/WT动物。我们的工作将CFTR-ΔF508兔添加到用于生物医学研究的CF动物模型工具库中。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5e7e/7862758/752e21a605cb/fgene-11-627666-g001.jpg

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