Allele suppression of an endogenous gene with antigens and nucleic acids that bind to polymorphisms within or adjacent to that gene so that one allele is preferentially suppressed

摘要

A method for suppressing expression of one allele of an endogenous gene is provided comprising providing suppression effectors such as antigens nucleic acids able to bind to polymorphisms within or adjacent to a gene such that one allele of a gene is exclusively or preferentially suppressed and if required a replacement gene can be introduced. The advantage is that the same suppression method when directed to polymorphisms could be used to suppress, in principle, many mutations in a gene. This is particularly relevant when large numbers of mutations within a single gene cause disease pathology.