摘要:
The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding cytochrome P450 (CYP) proteins. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. The invention also provides zinc finger nucleases that target chromosomal sequence encoding CYP proteins, as well as methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.
摘要:
Compositions and methods for identifying and/or modulating RNA transcripts and/or genes involved in fragile X syndrome and other associated disorders are provided. In particular, RNA targets for fragile X mental retardation protein (FMRP) have been identified by a novel monoclonal antibody to FMRP and a consensus sequence for the RNA binding region has been identified. Arrays for identifying compounds, proteins, nucleotides, and the like that modulate the RNA targets or associated genes are provided. Additionally, methods for modulating RNA targets are provided.
摘要:
Compositions and methods for identifying and/or modulating RNA transcripts and/or genes involved in fragile X syndrome and other associated disorders are provided. In particular, RNA targets for fragile X mental retardation protein (FMRP) have been identified by a novel monoclonal antibody to FMRP and a consensus sequence for the RNA binding region has been identified. Arrays for identifying compounds, proteins, nucleotides, and the like that modulate the RNA targets or associated genes are provided. Additionally, methods for modulating RNA targets are provided.