I came across a really cool application of CRISPR today, in Rob Stein’s NPR article, “First Sickle-Cell Patient Treated with CRISPR Gene-Editing Still Thriving.” If you’re interested, I highly suggest you give it a read — but I’ll summarize the findings here.

Victoria Gray, a 36-year-old mom and wife, volunteered for a clinical trial where doctors took cells out of her bone marrow, edited a gene in those cells using CRISPR, and then reinfused the modified cells back into her body. The idea of this method was that the edited cells would produce a protein called fetal hemoglobin, and alleviate sickle cell disease symptoms in a patient.

It has now been two years since Victoria was first injected with the modified cells, and she’s doing great. The experimental treatment has proven thus far to rid her of any symptoms of sickle cell disease, including sudden bouts of horrible pain, intensely debilitating fatigue, and intense pain medication.

In fact, she had a cold a few weeks ago — and she said that before her treatment, getting a cold could trigger one of the symptoms of her disease. Except this time, with the treatment, she didn’t end up having to go to the hospital; in fact, she said other than having a cold, she felt fine.

Today, doctors have treated over 45 patients with sickle cell or beta thalassemia, a related condition. The study reports that for 22 of those patients, the treatment is working. Dr. Haydar Frangoul, who works at the Sarah Cannon Research Institute in Nashville, Tennessee, leads the study. He said,

“It really changes patients’ lives…you see the change almost immediately after patients leave the hospital, where they are feeling better and able to resume their lives normally without the horrible complications that can happen.”

– Dr. Haydar Frangoul, Sarah Cannon Research Institute

CRISPR Therapeutics and Vertex Pharmaceuticals are collaborating to develop the treatment, and they hope to gain FDA Approval within the next year to two years. This is one example of CRISPR positively impacting patient’s lives due to gene-editing. Thanks for reading!