Contemporary Animal Models For Human Gene Therapy Applications

Area of Science:

• Biomedical Research
• Translational Medicine
• Gene Therapy

Background:

• Gene therapy has emerged as a significant treatment strategy for various diseases over the last 30 years.
• Successful human clinical trials for gene therapy have been reported since 2009.
• Preclinical animal models are essential for evaluating gene therapy vectors' safety, efficacy, dosage, and expression before human application.

Purpose of the Study:

• To review commonly used animal models in gene therapy research.
• To discuss the advantages and disadvantages of different animal models for preclinical gene therapy studies.
• To emphasize the importance of selecting appropriate disease-specific models for successful clinical translation.

Main Methods:

• Review of literature on animal models used in gene therapy.
• Categorization of animal models (murine, canine, non-human primates, rabbits, porcine, humanized mice).
• Discussion of model selection criteria based on study type and duration.

Main Results:

• Small animals (e.g., mice) are suitable for degenerative/aging studies due to shorter lifespans.
• Large animals (e.g., primates, pigs) are beneficial for longitudinal studies and dosage optimization.
• Humanized mice offer a more reliable platform for studying human tissues/cells in gene therapy.

Conclusions:

• Animal models are indispensable for in vivo testing of gene therapy vector technologies.
• Careful selection of disease-specific animal models is critical for assessing safety and efficacy.
• The choice between small and large animal models depends on the specific research needs and study design.