Adeno-Associated Viral Vectors and the Retina

  • John J. Alexander
  • William W. Hauswirth
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 613)

There are a variety of diseases of the retina arising from genetic and non-genetic causes, or a combination of both, that lead to the loss of vision. The retina is a prime location for gene therapy because of its accessibility, immune privileged status (Caspi, 2006), and susceptible cell types. Several strategies have been attempted to rescue retinal disease, including gene replacement (Acland et al., 2001), gene knockdown with both ribozymes (Gorbatyuk et al., 2007) and siRNA (Kiang et al., 2005), and therapeutic gene supplementation (Deng et al., 2005).


Gene Therapy rAAV Vector Ubiquitous Promoter Vitelliform Macular Dystrophy Gene Therapy Experiment 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.


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Copyright information

© Springer Science+Business Media, LLC 2008

Authors and Affiliations

  1. 1.Department of Molecular Genetics & MicrobiologyUniversity of Florida College of MedicineGainesville

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