Gene Editing: What it means for the future of medicine

BY ALEXANDER V. SHAHIN

Earlier this month, an international group of scientists convened at Washington, D.C. and gave the green light for human gene editing research, even on germline cells. However, in their statement, they warned researchers to be careful, highlighting the need for “appropriate regulatory oversight.”

This news represents a landmark decision for the future of human gene editing. In recent years, scientists have developed increasingly sophisticated techniques for manipulating nucleic acid. In particular, the CRISPR/Cas system has emerged as a powerful new tool that has made it possible to insert, delete, or alter DNA cheaper and quicker than ever before.

This system uses the enzyme Cas9, which bacteria usually produce to fight off viral infections. Recently, an alternative enzyme called Cpf1 was identified that may potentially prove to be an even more powerful tool in gene editing.

The thought of being able to modify human DNA in order to cure genetic diseases seems like something out of a sci-fi movie. The truth, however, is that genetic editing of the human body is already underway in patients. Recently, researchers at a company called Sangamo biopharmaceuticals announced that they would begin clinical trials using the CRISPR/Cas system to replace a mutant copy of the Factor IX gene in hemophiliacs. Earlier this year, a gene-editing tool known as TALENs was successful in treating a baby girl with leukemia. Furthermore, gene editing has already been used to engineer different crops and livestock.

There are many in the scientific community who remain skeptical of these new gene-editing technologies. Some researchers believe that tampering with an organism’s genome could have unforeseen, negative side effects. This is especially worrisome in germline editing, where any genetic change could be passed along to future generations. There are also many who question whether tinkering with the human genome is ethical.

These are all important questions that should be kept in minds as we move into the future of gene editing research. These new technologies have the potential to revolutionize medicine in the coming years, but it’s our responsibility to make sure it is used correctly. Of course, there is still a lot to learn before these technologies such as CRISPR/Cas become mainstream in clinical research.

However, the promises they have shown in their early days reveal a bright future for the field. We might be a long way off from living in a world of designer babies, but gene editing is already having a significant impact on people’s lives and will, hopefully, continue to do so in the near future.

 

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