Re-educating Our DNA

Back to the Genetic Home Reference. I have been seeing things that say gene therapy is one possible avenue of treatment for AMD and I have not been too sure what it is.

The Genetic Home Reference says gene therapy is the replacing of a mutated gene that causes disease with a ‘good’ copy of the gene. It may be used to deactivate a problematic gene or be introduced to help to fight a disease.

Because it is a new and potentially dangerous field of exploration, gene therapy is limited to diseases and conditions with no, known cures. That’s us, guys.

If you are not familiar with viruses, I offer a quick tutorial on how they work. Viruses are barebones ‘life’. There is debate as to whether or not they even meet criteria for being considered living organisms. Part of the definition of a living organism is that it can reproduce its own kind. Viruses cannot do that. Since viruses are no more than a strand of DNA wrapped in protein, a virus must ‘hijack’ a cell to make little viruses. The baby viruses eventually kill the host cell. This is why we get sick when we have a virus. Our cells have become factories/hosts for baby viruses. Sort of like the movie Alien in miniature. Yuck.

Since viruses have the ability to pierce cell membranes and mess with the cell’s ability to reproduce – effectively ordering the cell to reproduce a slew of baby viruses – it was decided they would be perfect for introducing ‘replacement parts’ into defective cell DNA.

The name for something that transfers things from one organism to another is called a vector. Therefore gene therapy uses viral vectors to get new DNA into cells. Researchers replace the basic, virus DNA with genetic material it hopes will repair the defective genes, then it ‘launches’ the viral vector to ‘attack’ and ‘hijack’ the cell.

How this relates to AMD is sort of basic. We know AMD is a genetically based condition. Change our genetics and we may eliminate the disease.

Johns Hopkins researchers have completed a phase 1 clinical trial during which they ‘infected’ retina cells with a virus carrying a gene that would cause the retina cells to produce a protein that interferes with the signals leading to the growth of new veins. The goal is to genetically alter enough cells they will produce enough of the protein to totally halt the neovascularization process and eliminate the need for shots.

Of course these studies are in very early days. One of the problems encountered was many people have developed resistance to the viruses use. They had met these enemies (the protein in the sheaths of the viruses) before and their defenses were in place. If the vectors are destroyed before they get to the targets, they cannot very well plant the new gene.

Still early days on gene therapy but it most definitely has promise. Remedial instruction to ‘re-educate’ our DNA may be on the way.

written July 16th, 2017

Next: Your Good Fortune

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