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In the simplest terms, gene therapy introduces genetic material into cells to compensate for abnormal genes or to make beneficial proteins.
The modified gene is delivered via a carrier, called a vector, either by an injection or intravenously directly into a specific tissue. The vector is taken up by individual cells and if the treatment is successful, the new gene delivered by the vector will make a functioning protein.
But this is not as easy as it sounds. Gene therapy has been around for decades and while many challenges have been overcome, researchers and scientists around the world are still having only limited success. Some of the bigger challenges include.
- Mass delivery and activation - the gene needs to be delivered to the right cells, in the right tissue, in the right quantity (which is several million cells). Once there the gene must be turned on to produce the proper protein. This is the biggest challenge facing gene therapy researchers.
- Immune response - the immune system is designed to battle intruders and therefore gene delivery vectors must be able to bypass the body's surveillance system. An immune response may cause severe or deadly complications.
- Missing the target - gene therapy is successful when the newly-introduced gene becomes a permanent part of the cell's genome by "stitching" itself into the cell's existing DNA. If the gene stitches itself in the wrong spot it can cause interruption in the function of a normal gene, leading to diseases such as cancer.
So what is the future of gene therapy?
If successful, gene therapy has the potential to cure millions of people of hereditary diseases that currently have very few treatment options. Last November (2012) the European Medicines Agency gave the Dutch biotech startup UniQure permission to sell its treatment. Although this is giving gene therapy a new boost of research efforts (and investors) it may be a few years before the treatment is approved in the U.S.
The UniQure treatment involves a gene needed for the body to break down fats; without it, patients can develop painful and even fatal inflammation of the pancreas. The treatment uses a modified version of the virus to deliver the gene therapy. Several other gene therapies, including treatments for early blindness, cancer, and neurodegeneration, are nearing the final stage of testing.
The completion of clinical trials is no guarantee that the technology will become approved or available in the U.S. but many companies and hospitals are prepping to begin manufacturing gene-based therapies.
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Re: The Future of Gene Therapy
