Viral vectors: from virology to transgene expression

Area of Science:

• Molecular Biology
• Biotechnology
• Medical Science

Background:

• Gene therapy, predicted for human application in the 1970s, has yet to become a standard medical practice despite some successes.
• The use of viral material for gene insertion (transgenic insertion) presents significant experimental and clinical hurdles.

Purpose of the Study:

• To review the challenges associated with employing viral vectors in gene therapy.
• To discuss the development and characteristics of major viral vector types used in gene therapy.

Main Methods:

• Review of existing literature on gene therapy, focusing on viral vector technology.
• Analysis of experimental and clinical problems encountered in gene therapy applications.
• Comparative discussion of key viral vector systems: retroviruses, adenoviruses, herpes simplex viruses, and adeno-associated viruses.

Main Results:

• Identified persistent experimental and clinical challenges hindering routine gene therapy application.
• Detailed the development and properties of prominent viral vectors crucial for gene delivery.

Conclusions:

• Overcoming the identified problems is essential for advancing gene therapy towards clinical routine.
• Understanding the nuances of different viral vector types (retroviruses, adenoviruses, HSV, AAV) is critical for successful gene therapy development.