Transgenic Animal Model
An animal that has been genetically modified to carry and express a foreign gene. Transgenic models can express human versions of drug targets, enabling more relevant preclinical testing of peptide compounds that may not interact with the animal's native receptor.
Technical Context
Transgenic models relevant to peptide drug development include: human receptor knock-in mice (expressing the human version of a drug target receptor — necessary when the peptide has species-specific receptor binding, e.g. human GLP-1R knock-in mice for more translatable GLP-1 RA pharmacology), human FGFR3 knock-in mice (modelling achondroplasia for vosoritide development), amyloid precursor protein (APP) transgenic mice (Alzheimer's disease models for neuroprotective peptide research), and tumor-bearing transgenic models (genetically engineered mouse models that spontaneously develop tumours expressing specific receptors). Transgenic models provide more clinically relevant pharmacology data than standard animal models when significant species differences exist in drug-target interactions. The development of humanised mouse models has been a significant advance for preclinical peptide drug evaluation.