Article
RNA based gene therapy for dominantly inherited diseases.
Current gene therapy - 1 Feb 2006
Pelletier Richard, Caron Solenne O P, Puymirat Jack
Abstract excerpt
There are numerous examples in the literature of gene therapy applications for recessive disorders. There are precious few instances, however, of studies conducted to treat dominantly inherited pathologies. The reasons are simple: there are fewer cases of dominantly inherited diseases on one hand, but mostly it is far easier to correct recessive mutations than dominant ones. Typically recessive mutations cause a...
Topics
- Animals
- Genes, Dominant
- Genetic Diseases, Inborn
- Genetic Therapy
- Humans
- Mutation
- RNA
- RNA Interference
- RNA, Antisense
- RNA, Catalytic