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Gene Therapy for Mouse Models of ADRP

  • Marina S. Gorbatyuk
  • William W. Hauswirth
  • Alfred S. Lewin
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 613)

Autosomal Dominant Retinitis Pigmentosa (ADRP) is caused by mutations in the rhodopsin gene (RHO) in 30% of patients, and dominant negative mutations are often associated with toxicity of the mutated protein. Preventing or retarding ADRP progression may require the repair or silencing of the defective RHO gene. Two strategies for RHO mRNA silencing have recently been proposed (Millington-Ward et al., 1997; Lewin and Hauswirth, 2001). They are based on the application of therapeutic molecules such as ribozymes (Rz) and small interfering RNA (siRNA) that inhibit RHO expression.

Keywords

Gene Therapy Retinitis Pigmentosa Retinal Degeneration Outer Nuclear Layer Dominant Negative Mutation 
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

  • Marina S. Gorbatyuk
    • 1
  • William W. Hauswirth
    • 1
  • Alfred S. Lewin
    • 2
  1. 1.Department of Molecular Genetics and MicrobiologyUniversity of Florida College of MedicineGainesvilleUSA
  2. 2.Departments of Molecular Genetics and the Center for Vision Science, Department of Molecular Genetics and MicrobiologyUniversity of Florida, University of Florida College of MedicineGainesvilleUSA

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