hnRNP A1 knockout animal model and use thereof

摘要

A nucleic acid construct comprising a genetic engineered heterogeneous nuclear ribonucleoprotein (hnRNP) A1 gene is provided. A transgenic mouse in which the expression of hnRNP A1 gene has been disrupted is also provided. The mouse is useful for studying the role of hnRNP A1 gene in normal and disease states of a developmental disorder and muscular diseases. Therefore, a method of screening a compound for potential use in prevention and/or treatment of developmental disorder and muscular diseases is further provided.

主权项

1. A transgenic knockout mouse the whose genome is manipulated to comprise a homozygous disruption of hnRNP A1 gene, wherein the mouse exhibits tongue atrophy as compared to a wild-type mouse in which the hnRNP A1 gene is not disrupted, wherein said homozygous disruption of hnRNP A1 gene comprising deletion of exons 2-8 of the hnRNP A1 gene, wherein the mouse exhibits no expression of hnRNP A1 gene, wherein the tongue atrophy comprises degenerated skeletal muscle cells.