Animal Models for Prenatal Gene Therapy: Rodent Models for Prenatal Gene Therapy

  • Jessica L. Roybal
  • Masayuki Endo
  • Suzanne M. K. Buckley
  • Bronwen R. Herbert
  • Simon N. Waddington
  • Alan W. Flake
Part of the Methods in Molecular Biology book series (MIMB, volume 891)


Fetal gene transfer has been studied in various animal models, including rabbits, guinea pigs, cats, dogs, and nonhuman primate; however, the most common model is the rodent, particularly the mouse. There are numerous advantages to mouse models, including a short gestation time of around 20 days, large litter size usually of more than six pups, ease of colony maintenance due to the small physical size, and the relatively low expense of doing so. Moreover, the mouse genome is well defined, there are many transgenic models particularly of human monogenetic disorders, and mouse-specific biological reagents are readily available. One criticism has been that it is difficult to perform procedures on the fetal mouse with suitable accuracy. Over the past decade, accumulation of technical expertise and development of technology such as high-frequency ultrasound have permitted accurate vector delivery to organs and tissues. Here, we describe our experiences of gene transfer to the fetal mouse with and without ultrasound guidance from mid to late gestation. Depending upon the vector type, the route of delivery and the age of the fetus, specific or widespread gene transfer can be achieved, making fetal mice excellent models for exploratory biodistribution studies.

Key words

Rodents In utero gene delivery Fetal gene therapy Mating Injection procedures Tissue and biological fluid sampling Biodistribution 


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Copyright information

© Springer Science+Business Media, LLC. 2012

Authors and Affiliations

  • Jessica L. Roybal
    • 1
  • Masayuki Endo
    • 1
  • Suzanne M. K. Buckley
    • 2
  • Bronwen R. Herbert
    • 1
  • Simon N. Waddington
    • 3
  • Alan W. Flake
    • 4
  1. 1.Department of Surgery, Children’s Center for Fetal Research and Center for Fetal Diagnosis and TreatmentChildren’s Hospital of PhiladelphiaPhiladelphiaUSA
  2. 2.Gene Transfer Technology Group, Institute for Women’s HealthUniversity College LondonLondonUK
  3. 3.Institute for Women’s Health, Gene Transfer Technology GroupUniversity College LondonLondonUK
  4. 4.Department of SurgeryChildren’s Hospital of PhiladelphiaPhiladelphiaUSA

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