Module 8 : GENE THERAPY : INTRODUCTION AND METHODS

Lecture 1 : Gene Therapy: Introduction and Methods

•  Adeno- Associated Virus (AAV)

Adeno-associated viruses (AAVs) are a group of small, single-stranded DNA viruses which cannot usually undergo productive infection without co-infection by a helper virus, such as an adenovirus.

Adeno-associated viruses are explained in detail in Module 5-Lecture 1.

•  Retroviral Vectors

Retroviruses are RNA viruses which possess a reverse transcriptase activity, enabling them to synthesize a complementary DNA. Following infection (transduction), retroviruses deliver a nucleoprotein complex (pre-integration complex) into the cytoplasm of infected cells. The viral RNA genome is reverse transcribed first and then integrates into a single site of the chromosome.

Figure 8-1.6.1.1c

Adaptedand modified from:

http://www.ncbi.nlm.nih.gov/books/NBK7570/figure/A2357/?report=objectonly