Enzo Biochem, Inc. has received a patent from the U.S. Patent and Trademark Office that covers nucleic acid compositions and methods for editing and correcting certain abnormalities in genes.
“Given the vast body of knowledge available through charting the human genome, the ability to correct inherent errors in the genes of a subject, and certain other medically important genetic material where alterations may be indicated, becomes very relevant,” said Dean L. Engelhardt, Ph.D. Senior Vice President of Enzo.
“The procedure outlined in the patent could provide the means for these corrections.” U.S. Patent No. 5,958,681 claims a method and materials suitable for correcting point mutations or small insertions or deletions of genetic material.
For example, genetic abnormalities caused by single nucleotide alterations or small deletions could be corrected through the insertion or exchange of the correct or desired sequences into the genome of the cell where the error exists.
Enzo said that it has begun discussions leading to possible further application of this technology towards specific therapeutic products.
“The fundamentals of the methods and materials covered in the patent have been recognized by researchers, and the `681 patent provides Enzo with a commanding position to pursue what appears to be a highly promising new genetic correcting mechanism,” said Dr. Engelhardt.
“This technology represents another novel approach for treating genetic defects and viral diseases utilizing Enzo’s pioneering science focused on modifying the genetic makeup of an organism.” The method and materials covered by the ‘681 patent work by correcting the specific gene within cells with defined short gene sequences.
These sequences are held in place through formation of a triple helix complex.
The principle of the invention is that when the short gene sequence containing the correct sequence is held in close proximity to the target DNA to be corrected, the probability of a double crossover or an editing event becomes quite high.
This involves triplex-forming DNA sequences that bind to specific regions in the DNA in a sequence-specific manner to provoke DNA repair.
The triple helix holds the sequences in close proximity and at a precise point in the gene long enough for recombination, exchange or insertion to take place with a high frequency.