Here is an article visible if you are at a university: http://www.ncbi.nlm.nih.gov/pubmed/18441370 http://www.ncbi.nlm.nih.gov/pubmed/18441370 In brief, this

Here is an article visible if you are at a university: http://www.ncbi.nlm.nih.gov/pubmed/18441370 http://www.ncbi.nlm.nih.gov/pubmed/18441370 In brief, this is a disease caused by an effective deficiency of a protein. It is theoretically possible to apply similar methods to other diseases of genetic deficiency. Other approaches are promising for diseases of overabundance or adverse gain of function mutations.