Methods for Treating HIV by Gene Therapy Using Ribozymes

Ribozymes are small catalytic RNAs with the ability to reversibly cleave covalent bonds in RNA in the complete absence of protein (1 ). With the exception of the RNA component of bacterial RNase P, presently characterized ribozymes catalyze a one-time intramolecular self-cleavage in their natural settings (1 –9 ). However, they can be engineered to act in trans in a truly catalytic manner to cleave multiple copies of a specific RNA target molecule. The first ribozyme to be identified was the self-splicing ribosomal RNA precursor of the protozoan Tetrahymena (2 ,3 ). Several other classes of RNA-cleaving RNAs have since been discovered (1 . Investigations of ribozymes have begun to yield diverse outcomes that range from novel hypotheses about pre-biotic evolution (7 ) to potential human gene therapies (10 ,11 )