re: Human Gene Therapy

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There are certain protocols involving stem cells in which the actual editing of your genomic data isn't done in vivo. If you weren't aware, we can essentially take any cell in the body and induce the formation of patient specific stem cells, which are known as inducible stem cells. This is done by exposing these cells to four specific transcription factors which effectively reprogram your cells. Once induced, the cells can be transfected with a viral cassette containing the therapeutic genes. If the gene is installed at the correct genomic coordinate, they can then be allowed to differentiate in vitro before being transplanted back into the donor.

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My body does not produce the Rs1 gene.

Retinoschisin is an extracellular protein that plays a crucial role in the cellular organization of the retina. This protein is assembled and secreted from photoreceptors and bipolar cells as a homo-oligomeric protein complex. Monomeric retinoschisin contains 224 amino acids with a leader sequence that is cleaved off upon preparation in the cell for secretion.

Mutations in this gene are responsible for X-linked retinoschisis an early-onset macular degeneration in males that results in a splitting of the inner layers of the retina and severe loss in vision.

So, you're saying a virus can help my body "kick start" and produce this gene?