Biotechnology / Gene Therapy

In gene therapy, a new gene is inserted into an adenovirus vector, which is then used to introduce modified DNA into a human cell. If the therapy treatment is successful, the new gene will end up taking root and making a functional protein.

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Gene therapy applications include the treatment, and hopefully, the curing of genetic and acquired diseases such as AIDS and cancer. Gene therapy can be used to target somatic (in which the genome of the recipient is altered, but this alteration isn’t passed on to the next generation) or gametes (i.e germline), in which the egg and sperm cells of the parents are altered for the purposes of passing on the modifications to their offspring.

Essentially, there are two basic ways of implementing gene therapy:

1. Ex vivo (outside the body) – using this method, cells from the patient’s blood or bone marrow are removed and grown in a lab. There, they are exposed to a virus that carries the desired gene. As the virus enters the cells, the desired gene becomes part of the cells’ DNA. The cells are grown in the lab before being returned to the patient via injection.

2. In vivo (inside the body) – using this method, vectors are used that deliver the desired gene to cells in the patient’s body.

Some of the challenges of gene therapy include:

1. High costs. Still a relatively new field of study, gene therapy is a pricey endeavor.

2. Our limited knowledge of the function of genes. In the grand scope of things, scientists know the functions of only a few genes, and even then the jury’s still out as to whether some or all genes have more than one function.

3. Gene delivery tools. As mentioned, vectors are used as gene carriers to insert genes into the body. The most common vectors used now are viruses, which – while they’re an effective means of gene transportation, can also introduce problems such as toxicity, immune responses, and gene control / targeting issues.

4. The challenge of multigene disorders and effect of environment. Fact is, most genetic disorders involve more than one gene, and furthermore, most diseases involve the interaction of several genes along with environmental factors. Cancer is a perfect example of a disease gene that can be inherited, while diet, smoking, exercise and other factors may be contributing factors to the disease as well.