Therapeutic siRNAs for dominant genetic skin disorders including pachyonychia congenita.

ABSTRACT: The field of science and medicine has experienced a flood of data and technology associated with the human genome project. Over 10,000 human diseases have been genetically defined, but little progress has been made with respect to the clinical application of this knowledge. A notable exception to this exists for pachyonychia congenita (PC), a rare, dominant-negative keratin disorder. The establishment of a non-profit organization, PC Project, has led to an unprecedented coalescence of patients, scientists, and physicians with a unified vision of developing novel therapeutics for PC. Utilizing the technological by-products of the human genome project, such as RNA interference (RNAi) and quantitative RT-PCR (qRT-PCR), physicians and scientists have collaborated to create a candidate siRNA th

SUBMITTER: Leachman SA 

PROVIDER: S-EPMC2587483 | biostudies-literature | 2008 Sep

REPOSITORIES: biostudies-literature