Genetic diseases spontaneous mutations

Heritable and spontaneous genetic disorders represent additional applications for therapeutic ribozymes targeting cellular genes. These include the beta-amyloid peptide precursor mRNA involved in Alzheimer s disease (Currie et al., 1997 Dolzhanskaya et al., 2000), and an autosomal-dominant point mutation in the rhodopsin mRNA that gives rise to photoreceptor degeneration and retinitis pigmentosa (Hauswirth and Lewin, 2000 LaVail et al., 2000). 

There are many reviews on constitutes a mouse model for human diseases (5-7), as well as models for specific diseases (8-22). It is beyond the scope of this chapter to discuss the pros and cons of each of these potential models, especially since a recent scan of the public literature and databases revealed over 1200 genetically engineered and spontaneous mouse mutations that have skin diseases that potentially serve as models for specific human diseases. Information on these are and where to find them is discussed below. Further, this chapter will not describe the usefulness of the mouse to study wound healing, and the reader is referred to other reviews on such (23, 24). 

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