Enzo Biochem Awarded Patent Covering Method to Correct
Certain Gene Abnormalities
Technology Would Permit "Editing" of DNA in a Cell
FARMINGDALE, N.Y.--(BUSINESS WIRE)--March 15, 2000-- Enzo
Biochem, Inc. (NYSE:ENZ - news)
announced today that it 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 this new technology provides the Company
with an additional therapeutic platform and
another unique approach for treating genetic-based diseases,
as well as other applications. Enzo also
said that this gene editing technique differs from other
gene therapies that usually require the insertion of
an entire new gene to correct a nonfunctional or incorrectly
functional gene.
This invention could lead to creation of a normally functioning
gene by correcting a mutated or altered
gene sequence. It could correct, for example, an inborn
error of metabolism for a condition such as
hemophilia. Potentially, it could also be used to correct
a mutated p53 gene that controls cell growth. In
the mutated gene, the cell growth is uncontrolled, resulting
in cancer. In the corrected p53 gene, the cell
growth would be restored to its normal, non-cancerous
state.
These genetic alterations could be used to inactivate oncogenes,
genes in a cancer cell that function to
maintain the cancerous state of the cell, or to inactivate
genes in a virus essential for continued growth of
the virus. Such genetic alterations could also have application
in genetic diseases such as Tay-Sachs
and sickle cell anemia.
In addition to its application in human therapeutics, this
new technology could find (I substituted d for e in
fine>>>HEY LOOK GUYS and GALS, IT WORKS!!!!!) use in veterinary
and agricultural applications.
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.
>>>END<<<
ENZO has harnassed a recombinant trick (mechanism) of the
recombinant virus, to INTEGRATE a repair,
rather than alter the human genome, as a retro-virus can
successfully initiate. Very ingenious!
Looks like a very strong Patent. Note the gene seeking
attribute. If a specific "FIX" is targeted to a
sequence just long enough to match only one in the human
genome, the insertion, utilizing perhaps a
lentiviral vector, which carries ONLY this payload, would
repair ONLY THOSE CELLS with the need for
the repair.
So you take HIV, strip out its payload for manufacturing
more HIV, replace instead the specific construct
in question and deliberately infect the Patient. Sounds
bad? Well, once HIV has no payload it cannot pull
one from thin air. It ceases to be HIV and instead becomes
HRV (Human Repair Virus). Now that would
be wonderful! And given ENZO's track record with stealth
techniques, I expect a package with no
measurable presence, save the fix!
Could it be that this Patent is so well conceived and timed,
that the infringers of old NEED IT BADLY? So
ENZO gets to set its terms, which include a reasonable
BACK PAYMENT, to pony up and share this
wonder? Could be?
Sincerely,
Larry Glaser
P.S. Trioxynucleic Acid is born...