Within the past few decades, as we have begun to unlock the way in which human beings have been engineered through our DNA, researchers have also started to explore how we can work with genetic solutions to cure what ails us. Up until this point, those with rare diseases often had little recourse. But recent research has put us on the precipice of a new and exciting time—a time when people may be cured by some editing of their own genes.

 

Offering Hope for Cystic Fibrosis Sufferers and More

With editing technology becoming more and more advanced with each passing day, patients who suffer from both polygenic and monogenic disorders may now see some hope on the horizon. It seems that even certain cancers and heart diseases will now be addressed through gene therapy. Before, there was nothing that the medical community could do for people who were suffering from these disorders; for most of them, there weren’t even trials that patients could sign up for if they wanted.

 

Viral Vectors Will Make the Difference

Along with gene editing advances, researchers are also extremely bullish on the delivery techniques that will be used to introduce changes to the human body. Right now, they are using lentivirus with stem cells, as well as adeno-associated virus (AAV) as the primary delivery methods. At a recent Boston conference centered around a discussion of rare disorders, many of the nation’s top researchers seemed thrilled that finally progress was being made in this arena. AAV vectors, in particular, hold a lot of promise for many different types of applications with patients, but obviously, a great deal of research will need to be done before the general population will see such treatments being offered.

 

Laying the Groundwork for Progress

Everyone involved with gene therapy agrees that it will take some time. However, there is a huge push from government agencies to make these transformations happen sooner rather than later. Allocating hundreds of millions of dollars towards funding, the National Institutes of Health has proven to be a critical resource during this time of discovery. For-profit medical companies are also getting into the action, as it will undoubtedly become a highly profitable sector in the years to come.