What are we going to see from gene and cell therapy in the near future?

Future applications  of gene therapy:

Gene therapy is being used in many ways. For example, to:

• Replace missing or defective genes;
• Deliver genes that speed the destruction of cancer cells;
• Supply genes that cause cancer cells to revert back to normal cells;
• Deliver bacterial or viral genes as a form of vaccination;
• Provide genes that promote or impede the growth of new tissue; and;
• Deliver genes that stimulate the healing of damaged tissue.

A large variety of genes are now being tested for use in gene therapy. Examples include: a gene for the treatment of cystic fibrosis (a gene called CFTR that regulates chloride); genes for factors VIII and IX, deficiency of which is responsible for classic hemophilia (hemophilia A) and another form of hemophilia (hemophilia B), respectively; genes called E1A and P53 that cause cancer cells to undergo cell death or revert to normal; AC6 gene which increases the ability of the heart to contract and may help in heart failure; and VEGF, a gene that induces the growth of new blood vessels (angiogenesis) of use in blood vessel disease.

A short synthetic piece of DNA (called an oligonucleotide) is being used by researchers to "pre-treat" veins used as grafts for heart bypass surgery. The piece of DNA seems to switch off certain genes in the grafted veins to prevent their cells from dividing and thereby prevent atherosclerosis.

Future applications  of  cell Therapy

Advancements in stem cell therapy have been gaining steam over the last decade. This is a promising indication that adult and embryonic stem cells will be used in the coming decade to treat and possibly cure cancer, paralysis, cardiac failure, stroke, type 1 diabetes mellitus, and many more. Recent breakthroughs in cellular and molecular biology have widened the scene in which new stem cells can be developed and used alongside existing clinical approaches. In the near future, hospitals and clinics will be able to regenerate a patient's tissue, ranging from their own heart muscle, lung, liver, and kidney tissue, eyes, even hair. They will also be able to help people with baldness, missing teeth, deafness, blindness, birth defects, wound healing, and infertility.

For example, research conducted in 2012 to repair spinal cord damage in rats and mice led to partial recovery in motor function after SC injections [8]. Similar models with stroke have led to a similar functional recovery after mesenchymal stem cells (MSCs) were injected into the carotid artery. But, just to illustrate the difficulties researchers face, when cells were injected intravenously to repair damaged brain tissue, results showed a substantially diminished cellular uptake, possibly due to the blood-brain barrier .

Another difficulty is that if and when iPS cells are reprogrammed using traditional methods, either with a virus or episome vector, the residual foreign proteins it contains elicits an immune response to destroy the stem cells. So, until an improved vector can be developed, researchers have proposed using MSCs as an alternative for therapy due to their ability to successfully mitigate the immune response. However, the limited number of trials using MSCs and difficulty producing a sufficient quantity of cells are the current limitations to a widespread application.

The reported successes of cell therapy in patients are due, only in part, to the regeneration of cells after injection. The primary mechanism is really the ability of SCs to first repair the existing tissue, which will ultimately improve organ function.

So, while a number of advancements are clearly being made, cell therapy is a dynamic process, and deviating from the precise procedure in any one step, such as how stem cells are produced and cultured, how cells are handled, or how they are injected, can all lead to significant statistical variations in clinical tests .

Right now, there approximately 3,400 controlled clinical trials registered as Cell Therapy in the NIH database (http://clinical-trials.gov/). In total, almost 1,900 trials have finished, although only about 125 have presented their results. Less than 50 trials are currently focused on endocrinology.

It is obvious to many that we not only want but need improvements to the applications of cell therapy. Cooperation between the private and public sectors will be important for applying stem cell technologies to improve cell therapy in the future. Traditional and progressive approaches will need to be used to promote such advancements.