Certain genetic disorders are caused by mutations in a single gene, such as cystic fibrosis and sickle cell anemia. The field of genome editing looks to correct these mutations either by inserting non-mutated copies of these genes or removing them from the genome entirely. Such manipulations would effectively cure these diseases, a goal that gene therapy companies have been pursuing for the past couple decades.
“There has been so much investment in making this technologically feasible for the last twenty years,” said Katrine Bosley, CEO of Editas Medicine. Despite encountering safety concerns early on, genome editing is now on the cusp of making a true difference for patients. Gene therapy has been used successfully on a small scale to cure specific forms of genetic blindness and is now being expanded to address even more disorders.
“You see data that is transformative to patients,” said Bosley of these new applications. Instead of receiving monthly or even daily treatments, patients undergoing gene therapy will only require a single genetic manipulation. After that, the patient’s new, healthy DNA will behave normally. Such technologies have the power to create enduring benefits and even completely cure certain genetic disorders.
Lea is a PhD student at MIT studying molecular and cellular neuroscience. She is interested in translational disease research and is currently examining the role of neuronal identity in Huntington's Disease. Lea graduated from Harvard with an AB in neurobiology and has interned with biotech startups in neuropharmacology and biomedical engineering.
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