Vector design Tour de Force: integrating combinatorial and rational approaches to derive novel adeno-associated virus variants

Mol Ther. 2014 Nov;22(11):1900-9. doi: 10.1038/mt.2014.139. Epub 2014 Jul 22.

Abstract

Methodologies to improve existing adeno-associated virus (AAV) vectors for gene therapy include either rational approaches or directed evolution to derive capsid variants characterized by superior transduction efficiencies in targeted tissues. Here, we integrated both approaches in one unified design strategy of "virtual family shuffling" to derive a combinatorial capsid library whereby only variable regions on the surface of the capsid are modified. Individual sublibraries were first assembled in order to preselect compatible amino acid residues within restricted surface-exposed regions to minimize the generation of dead-end variants. Subsequently, the successful families were interbred to derive a combined library of ~8 × 10(5) complexity. Next-generation sequencing of the packaged viral DNA revealed capsid surface areas susceptible to directed evolution, thus providing guidance for future designs. We demonstrated the utility of the library by deriving an AAV2-based vector characterized by a 20-fold higher transduction efficiency in murine liver, now equivalent to that of AAV8.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Amino Acid Sequence
  • Amino Acids
  • Animals
  • Capsid Proteins / genetics*
  • DNA, Viral / analysis*
  • Dependovirus / genetics*
  • Gene Library
  • Genetic Therapy
  • Genetic Vectors / administration & dosage*
  • HEK293 Cells
  • High-Throughput Nucleotide Sequencing
  • Humans
  • Liver / virology*
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Organ Specificity
  • Sequence Analysis, DNA
  • Transduction, Genetic

Substances

  • Amino Acids
  • Capsid Proteins
  • DNA, Viral