Human Gene Therapy

Rats are one thing.


Doing it in humans is another.


IMO the big thing holding it back is the massive amount of regulation of human testing. Who would want to volunteer for genetic testing that could give you super aids cancer?

The good news is that it still seems very promising. The bad news is that we are still far away from making it commonplace. Every small step takes years of work.

These things need more time and more money.

Do you keep up with clinical trials? See if you are eligible?

quote:

---

Do you keep up with clinical trials? See if you are eligible

---

I've tried everything.

University of Michigan is the only system that really has done any trials.

I tried the acid eye drops, they helped somewhat.

But, my goodness, the pain, with very little improvement.

My best bet may be Europe.

quote:

---

Thank the FCC

---

You mean FDA?

The lawyers hamper positive movement.

I'm sorry , but if we are going to move forward, there will be some sad souls that are hurt.

I'm willing to be one for the advancement of the health of mankind. I'm not talking about something completely foolish, but I understand people may be hurt.

I just don't want to do nothing.

Have you read about the Human Microbiome Project?

LINK

quote:

---

Examinations of DNA sequences served as the equivalent of an old-time microscope, said Curtis Huttenhower of the Harvard School of Public Health, an investigator for the microbiome project. They allowed investigators to see — through their unique DNA sequences — footprints of otherwise elusive bacteria.

---

Eta: Doubt this helps with going blind, but it's somewhat relevant.

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 to a pluripotent state.

Once induced, the cells can be transfected with a viral cassette containing the therapeutic gene. 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.

This protocol was used a few years ago to effectively cure mice afflicted with sickle cell anemia.

quote:

---

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.

---

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?