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TRANSGENIC ANIMAL MODEL FOR THE DEVELOPMENT OF NOVEL THERAPEUTIC APPROACHES

机译:开发新型治疗方法的转基因动物模型

摘要

The invention relates to a non-human transgenic animal model which contains a directed mutation in the gene coding for muscle 6-phosphofructo-1-kinase. The inventive method of producing the non-human transgenic animal comprises the following steps consisting in: i) creating a recombination construction ii) transfecting the recombination construction into pluripotential cells iii) selecting the cells transfected in step (ii) which express the aforementioned marker(s) iv) introducing said transfected cell into an embryo in the blastocyte stage v) transferring the manipulated blastocytes to recipient females in order to obtain chimeric mice vi) crossing the chimeric animals with control animals in order to produce a heterozygous animal for mutation and, subsequently, vii) crossing the heterozygous animals obtained in (vi) in order to obtain homozygous animals for mutation in the gene that codes for muscle PFK1. Said transgenic animal model can be used in the development of novel therapeutic approaches for the treatment of diseases such as glycogenosis, type II diabetes and Alzheimer's.
机译:本发明涉及一种非人转基因动物模型,该模型在编码肌肉6-磷酸果糖-1-激酶的基因中含有定向突变。生产非人类转基因动物的本发明方法包括以下步骤,包括:i)建立重组构建体ii)将重组构建体转染到多能细胞中iii)选择在步骤(ii)中转染的表达上述标志物的细胞(i) s)iv)将所述转染的细胞引入胚细胞阶段的胚胎中v)将经操纵的胚细胞转移至受体雌性以获得嵌合小鼠vi)将嵌合动物与对照动物杂交以产生用于突变的杂合动物,以及随后,vii)杂交在(vi)中获得的杂合动物,以获得用于编码肌肉PFK1的基因突变的纯合动物。所述转基因动物模型可用于开发新颖的治疗方法以治疗疾病,例如糖原病,II型糖尿病和阿尔茨海默氏病。

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