Animal Models in Retinoblastoma Research

  • Thomas A. Mendel
  • Anthony B. DanielsEmail author


Many animal models of retinoblastoma exist, each of which recapitulates some, but not all, of the features of human disease. The most commonly used models that are available are described in this chapter. These include various genetic and xenograft models of retinoblastoma and include a multitude of species, such as mice, rats, rabbits, pigs, and nonhuman primates. A wide array of uses of animal models in retinoblastoma research are presented here, including those for the study of tumor biology and response to treatment, assessment of pharmacokinetics, and measurement of chemotherapeutic toxicity and efficacy for drug discovery. Importantly, we discuss the relative strengths and limitations of each model. Factors for investigators to consider in designing various types of experiments are highlighted, including type of tumor model (genetic or xenograft), immune status (native, immunologically naïve, genetically immunocompromised, or pharmacologically immunosuppressed), and choice of species. While a particular model may be ideal for a certain use, no single model is appropriate for all experiments. Species-specific factors are especially critical when studying regional and local chemotherapy delivery.


Retinoblastoma Animal model Pharmacokinetics Safety Knockout Xenograft Drug discovery 


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© Springer Nature Switzerland AG 2019

Authors and Affiliations

  1. 1.Department of Ophthalmology and Visual SciencesVanderbilt Eye InstituteNashvilleUSA
  2. 2.Vanderbilt-Ingram Cancer CenterVanderbilt University Medical CenterNashvilleUSA

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