AAV-based vectors for human diseases modeling in laboratory animals

Area of Science:

• Biotechnology
• Genetics
• Virology

Background:

• Therapeutic drug and vaccine development necessitates accurate animal models of human diseases.
• Both native and transgenic animals serve as models, with transgenic animals offering tailored properties.
• Rapid generation of transgenic models is crucial, particularly during health crises like pandemics.

Purpose of the Study:

• To analyze the features of the adeno-associated virus (AAV) genome that make it a suitable vector for transgenesis.
• To review the application of AAV vectors in creating animal models for various human diseases.

Main Methods:

• Review of scientific literature on adeno-associated virus vectors and transgenesis.
• Analysis of the adeno-associated virus genome structure and function for gene delivery.
• Compilation of examples demonstrating the use of AAV vectors in disease modeling.

Main Results:

• The adeno-associated virus genome possesses advantageous characteristics for use as an expression cassette in gene delivery.
• AAV vectors have been successfully employed to generate animal models for hereditary, oncological, and viral human diseases.
• The efficiency and versatility of AAV vectors facilitate the rapid production of essential research models.

Conclusions:

• Adeno-associated virus vectors are a powerful tool for efficient transgenesis and the rapid development of disease-specific animal models.
• The application of AAV vectors accelerates research in areas including genetic disorders, cancer, and infectious diseases.
• AAV-mediated transgenesis is vital for advancing preclinical research and the development of novel therapeutics and vaccines.