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NON-HUMAN MODEL OF GESTATIONAL AND ADULT FOLATE DEFICIENCY AND USES THEREOF

机译:生殖和成人叶酸缺乏症的非人类模型及其应用

摘要

Disclosed is a genetically modified non-human animal that is a model for defective folate transport. Specifically the non-human animal comprises a genetic modification that results in decreased or absent expression and/or biological activity of the endogenous reduced folate carrier. The animal can be further modified by introduction into the genome of a nucleid acid molecule encoding the human reduced folate carrier. Such an animal can be used in methods to identify compounds useful for treating a variety of conditions associated with defective folate transport, or for the identification of anti-folate compounds useful for treating cancer. The animal can also be used to study the biochemical and molecular events associated with folate transport. Compounds identified using such a model are valuable for therapeutic treatments.
机译:公开了一种转基因的非人类动物,其是叶酸运输缺陷的模型。特别地,非人类动物包括导致内源性还原叶酸载体的表达降低和/或缺乏和/或生物学活性的基因修饰。可通过将编码人还原叶酸载体的核酸分子引入基因组来进一步修饰动物。这样的动物可用于鉴定可用于治疗与叶酸运输缺陷有关的多种病症的化合物,或用于鉴定可用于治疗癌症的抗叶酸化合物的方法。该动物还可用于研究与叶酸转运相关的生化和分子事件。使用这种模型鉴定的化合物对于治疗是有价值的。

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