Peer-reviewed veterinary case report
A transgenic mouse model for gene therapy of rhodopsin-linked Retinitis Pigmentosa.
- Journal:
- Vision research
- Year:
- 2008
- Authors:
- O'Reilly, Mary et al.
- Affiliation:
- Smurfit Institute of Genetics
Abstract
Mutational heterogeneity in genes causative of dominantly inherited disorders represents a significant barrier for development of therapies directed towards correction of the primary genetic defect. To circumvent the mutational heterogeneity present in rhodopsin- (RHO-) linked autosomal dominant Retinitis Pigmentosa (adRP), a strategy involving suppression and replacement of RHO has been adopted. RNA interference- (RNAi-) mediated suppression of RHO has been explored as has the generation of an RNAi-resistant replacement gene using the degeneracy of the genetic code. Additionally, the functional equivalence of codon-modified replacement genes has been demonstrated in a transgenic animal (RHO-M). Suppression and replacement, while exemplified by adRP, may also be relevant to many other dominantly inherited diseases with the hallmark of mutational heterogeneity.
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Search related cases →Original publication: https://pubmed.ncbi.nlm.nih.gov/17920651/