Transgene

Transduction (biophysics)

Virus

Adeno-associated virus

Duchenne muscular dystrophy

Biology

Gene

Genetic enhancement

Gene delivery

Virology

Capsid

Recombinant DNA

Vector (molecular biology)

Viral vector

Tropism

Genetics

Biochemistry

Nature Reviews Genetics

Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic effects have been achieved for other rare diseases, including haemophilia and Duchenne muscular dystrophy. However, current research indicates that the genetic modification of AAV vectors may further facilitate the success of AAV gene therapy. Vector engineering can increase AAV transduction efficiency (by optimizing the transgene cassette), vector tropism (using capsid engineering) and the ability of the capsid and transgene to avoid the host immune response (by genetically modifying these components), as well as optimize the large-scale production of AAV. Adeno-associated virus (AAV) vector-mediated gene delivery has had long-term therapeutic effects for several diseases, including haemophilia and Duchenne muscular dystrophy. Genetically modifying AAV vectors to increase their transduction efficiency, vector tropism and ability to avoid the host immune response may further increase the success of AAV gene therapy.

Engineering adeno-associated virus vectors for gene therapy