Patent ID: 6077705
Filing Date: 2000-06-20
Classification: A61K,C12N

Abstract:
A bifunctional expression vector for replacement of a mutant gene, said vector comprisinga regulatory cassette encoding a site-specific hammerhead ribozyme which degrades the mRNA of said mutant gene at a GUX triplet site within a defined region of the mRNA, wherein X is selected from the group consisting of C, U, and A, said defined region comprising said GUX triplet site and two flanking regions flanking said GUX triplet site, said hammerhead ribozyme comprising a structural domain interposed between two complementarity regions which are complementary to said two flanking regions, wherein each of said two complementarity regions has a length of at least 10 nucleotide residues, and wherein the combined nucleotide sequence of said structural domain and said two complementarity regions is selected from the group consisting of SEQ ID NOs: 13, 15, 17, 19, 21, and 23; anda structural cassette comprising a normal copy of said mutant gene, wherein the nucleotide sequence of said defined region of the mRNA of said normal copy differs from the nucleotide sequence of said defined region of the mRNA of said mutant gene, whereby said hammerhead ribozyme does not degrade the mRNA of said normal copy.