Adeno-Associated Virus Methods and Protocols

Today, progress in rAAV-mediated gene transfer is so robust that long-term, efficient, and regulatable transgene expression is reproducibly achieved in large animal models. The complexity of  gene transfer agents in the context of their clinical use requires investigators from a wide variety of back...

Full description

Bibliographic Details
Other Authors: Snyder, Richard O. (Editor), Moullier, Philippe (Editor)
Format: eBook
Language:English
Published: Totowa, NJ Humana 2011, 2011
Edition:1st ed. 2011
Series:Methods in Molecular Biology
Subjects:
Online Access:
Collection: Springer eBooks 2005- - Collection details see MPG.ReNa
Table of Contents:
  • Adeno-Associated Virus Biology
  • Design and Construction of Functional AAV Vectors
  • AAV Capsid Structure and Cell Interactions
  • Exploiting Natural Diversity of AAV for the Design of Vectors With Novel Properties
  • Gene Therapy in Skeletal Muscle Mediated by Adeno-Associated Virus (AAV) Vectors
  • AAV-Mediated Liver-Directed Gene Therapy
  • Recombinant AAV Delivery to the Central Nervous System
  • AAV Mediated Gene Therapy for Retinal Degenerative Diseases
  • Adeno-Associated Virus Vector Delivery to the Heart
  • Evaluation of the Fate of rAAV Genomes Following in vivo Administration
  • Measuring Immune Responses to Recombinant AAV Gene Transfer
  • Modification and Labeling of AAV Vector Particles
  • AAV-mediated Gene Targeting
  • Preclinical Study Design for rAAV
  • Biodistribution and Shedding of AAV Vectors
  • Production and Purification of Recombinant Adeno-Associated Vectors
  • rAAV Vector Product Characterization and Stability Studies
  • rAAV Human Trial Experience