Article
Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2006
Tessitore Alessandra, Parisi Fabiana, Denti Michela Alessandra, Allocca Mariacarmela, Di Vicino Umberto, Domenici Luciano, Bozzoni Irene, Auricchio Alberto
Abstract excerpt
Autosomal dominant retinitis pigmentosa caused by the frequent rhodopsin P23H mutation is characterized by progressive photoreceptor cell death eventually leading to blindness and for which no therapies are available. Considering the gain-of-function effect exerted by the P23H mutation, strategies aimed at silencing the expression of the mutated allele, like RNA interference, are desirable. We have designed small...
Topics
- Alleles
- Animals
- Animals, Genetically Modified
- Base Sequence
- Dependovirus
- Gene Silencing
- Genetic Vectors
- Mice
- Models, Animal
- Molecular Sequence Data
- Mutation
- Proline
- RNA, Small Interfering