Gene Therapy

It seems to me….

We need 10,000 genomes, not 100, to start to understand the link between genetics, disease, and wellness.” ~ Craig Venter[1].

Medical progress, as in all science, advances based on the work of predecessors. Researchers publish experimental results in peer-reviewed scientific papers recording processes used which are then validated by still other researchers. While effective in most fields, there are increasing reports of scientists being unable to replicate published results in biomedical research and its application to therapeutic intervention in the treatment of diseases. In many ways, this is a characteristic of progress indicating that science is advancing beyond the simpler least multifaceted research. Biology is complex and processes work neither linearly nor independently but in tightly related interconnected networks. Attempting to understand the workings of the human cell and apply that understanding to the treatment of illness and disease is exceptionally difficult.

RNA interference research, a mode of gene regulation, began only about ten years ago. Little is yet known about the inner workings of human cells and given the complexity and uncertainty, it is obvious why identical experiments can produce differing results[2].

DNA (deoxyribonucleic acid), RNA (ribonucleic acid), and proteins, along with noncoding DNA sequences, are the primary actors in the drama of life. DNA encodes genetic information, proteins support life’s processes, and RNA functions as the messenger between them.

Noncoding DNA sequences, sometimes referred to as “junk DNA”, are components of an organism’s DNA that do not encode protein sequences. Noncoding sequences have likely, but as yet mostly undetermined, functions. Many noncoding sequences are functional including genes for functional RNA molecules and sequences such as origins of replication, centromeres, and telomeres. Over 98 percent of the human genome is noncoding DNA. As most human DNA is shared with non-humans, much of what determines us as a species most likely is contained in these noncoding sequences.

The majority of medication available today function by manipulating proteins either by altering the amount produced or by blocking their function. Not all proteins, some of which constitute desirable targets, have been susceptible to this type of manipulation – their active sites are located within narrow channels or they are part of the cell’s internal skeleton[3].

Gene therapy is the use of DNA as a drug to treat disease by delivering therapeutic DNA into a patient’s cells. Its most common form involves using DNA that encodes a functional therapeutic gene to replace a mutated gene. While conceptualized in 1972, the first FDA-approved gene therapy experiment in the U.S. did not occur until 1990, and the first gene therapy treatment to be approved for clinical use in either Europe or the U.S. was just in 2012.

Scientists have long been aware of RNA’s basic housekeeping role within the cell but only within the last few years have researchers discovered new forms of RNA that could result in more precisely targeted medical treatments. These types of RNA that are able to direct specialized proteins to block or even totally silence certain cellular processes are enabling researchers to develop new, more precise medical procedures[4].

There now is investigation into whether these new forms of RNA are able to utilize noncoding sequences to also function as genetic manipulators. An entirely new world of medical progress appears possible.

That’s what I think, what about you?

[1] John Craig Venter is an American biotechnologist, biochemist, geneticist, entrepreneur, and one of the first to sequence the human genome.

[2] Kiermer, Veronique. Eureka Once, Eureka Twice, Scientific American, May 2014, p13.

[3] Gorman, Christine, and Dina Fine Maron. The RNA Revolution, Scientific American, April 2014, pp52-59.

[4] Gorman, Christine, and Dina Fine Maron. RNA Shines In New Roles, Scientific American, April 2014, p55.

About lewbornmann

Lewis J. Bornmann has his doctorate in Computer Science. He became a volunteer for the American Red Cross following his retirement from teaching Computer Science, Mathematics, and Information Systems, at Mesa State College in Grand Junction, CO. He previously was on the staff at the University of Wisconsin-Madison campus, Stanford University, and several other universities. Dr. Bornmann has provided emergency assistance in areas devastated by hurricanes, floods, and wildfires. He has responded to emergencies on local Disaster Action Teams (DAT), assisted with Services to Armed Forces (SAF), and taught Disaster Services classes and Health & Safety classes. He and his wife, Barb, are certified operators of the American Red Cross Emergency Communications Response Vehicle (ECRV), a self-contained unit capable of providing satellite-based communications and technology-related assistance at disaster sites. He served on the governing board of a large international professional organization (ACM), was chair of a committee overseeing several hundred worldwide volunteer chapters, helped organize large international conferences, served on numerous technical committees, and presented technical papers at numerous symposiums and conferences. He has numerous Who’s Who citations for his technical and professional contributions and many years of management experience with major corporations including General Electric, Boeing, and as an independent contractor. He was a principal contributor on numerous large technology-related development projects, including having written the Systems Concepts for NASA’s largest supercomputing system at the Ames Research Center in Silicon Valley. With over 40 years of experience in scientific and commercial computer systems management and development, he worked on a wide variety of computer-related systems from small single embedded microprocessor based applications to some of the largest distributed heterogeneous supercomputing systems ever planned.
This entry was posted in Biology, Biology, Centromeres, Deoxyribonucleic Acid, Disease, DNA, Gene Therapy, Genes, Genetics, Genomes, Health, medical, medicine, Proteins, Research, Ribonucleic Acid, RNA, Science, Telomeres and tagged , , , , , , , , , , , , , , , , . Bookmark the permalink.

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