Gene Therapy had a Rocky Start but its Promise for Rare Diseases Holds Strong

During the 1990s, researchers had a dream that by 2020, every individual would have access to their own genome. This would aid in their diagnosis and treatment as soon as they started feeling that something was off.

However, there was a problem with this dream. Not all genetic diseases are the result of a single mutation. Most, in fact, are a conglomeration of mutations in a few different genes. These mutations, in combination with environmental and other factors, cause the disease to occur. This means they can’t be cured with a single genetic fix.

In the 80s-90s, many researchers believed the contrary. They associated a wide array of common diseases with a single mutation. However, follow up studies eventually eliminated this hope.

That said, some rare diseases do have the potential to be cured by gene therapy.

Rare Diseases

For example, Huntington’s disease is caused by a single gene mutation. This means there is potential for it to be cured with gene therapy. This procedure essentially replaces the gene causing the mutation with a normal copy.

So far, gene therapy has made significant progress, and for rare diseases caused by a single mutation, researchers are hopeful about its potential.

Unfortunately, the process is expensive. This is made harder due to the fact that it is typically only an option for conditions which have a very small population of patients. This makes finding funding difficult. But that doesn’t mean researchers aren’t stopping their studies. In fact, they’re making steady strides.

The human genome project is helping to make this inquiry possible.

The Human Genome Project

This project was a result of international collaboration among scientists.

Together, this team has mapped and sequenced all human DNA. Beginning in 1990, this project has brought hope to many.

The goal is that it will help to advance the study of DNA sequencing, along with its manipulation. While at first the hope was that it would lead to a cure for many common diseases, researchers have revised their vision.

Unfortunately, we have learned the hope of common genetic cures which was so widespread in the 90s is not a realistic feat. Nevertheless, gene therapy research continues to bring rare disease patients hope.

You can read more about gene therapy research here.

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