United States: Gene therapy involve the introduction of genetic material, DNA, or RNA, to manage the state of progression of a disease. It is usually the process of entering genetic material into a person’s cells to correct a defective or missing gene.
Despite these treatments showing evidence of success in the expression of the therapeutic gene in the target tissue, they have also had profound toxic effects.
As reported by the medicalnewstoday, these side effects ranged from an overactive immune system, the wrong positioning of the genes in the human gene map thus activating genes that cause cancer, right up to death.
Advancements in technology have seen an enhancement of delivery systems that get the therapeutic genes to the target tissue and thus the progress of gene therapies by and large that of retinal diseases.
The eye’s retina is easily accessible hence the practical target for gene therapy. In addition, the ocular barriers, which includes the eyeballs, are sequestered from the rest of the body by physical barriers that confine the immune response in the eye.
The ocular barriers also limit the distribution of the vectors to the eyes and thus limit the show of the therapeutic gene in other body parts.
It is has been as a result of gene therapy that many inherited retinal diseases that are considered untreatable can be treated. Other than these kinds of genetic retinal diseases, there is evidence that gene therapy notions are effective in acquired retinal disease, which has got diverse etiology including glaucoma.
In what ways are gene therapies for eye conditions administered?
Theindices for the development of gene therapy for retina diseases involve various factors connected with the kind of delivery system and the way of administration.
Such considerations pertain to the size of the therapeutic gene, the type of target retinal cell, the immunogenicity of the vector carrier and cost factors. Gene therapies require the presence of viable target cells for their success and their efficacy starts to decline with the progressive degeneration of the tissue.
Gene therapy for retinal disease involves the administration of the genetic to the retina using a vehicle or vector, Vectors are genetically modified agents used to deliver genetic material such as a functional copy of the mutated gene to the cell.
While all the viruses are the most commonly used the vectors for the retinal disease, non-viral gene transfer methods are also being investigated.
Viral vector delivery systems.
Genetically modified viruses, including the adenoviruses, lentivirus and the adeno-associated viruses and have been used as the vectors for the retinal diseases.
These viral vectors can be genetically modified to selectively express the therapeutic gene in the target tissues or reduce the immune response to the virus.