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Treatment

CRISPR Gene Editing Continues to Benefit the First Sickle Cell Patient to Receive the Treatment

For roughly a decade, the technology known as CRISPR gene editing has been dominating headlines in the fields of genetics. An acronym for “clustered regularly interspaced short palindromic repeats,” CRISPR is an incredibly fast, easy, and precise method for editing segments of DNA.

The revolutionary tool constitutes a tremendous step forward for clinical genetic research. It has already led to some amazing advancements in the fight against hereditary diseases that range from cystic fibrosis to Marfan syndrome. With the potential to aid in the prevention and treatment of cancer among other common terminal illnesses, CRISPR’s tremendous promise shows few limitations.

One of the first people to find her life transformed by the power of CRISPR gene editing, Victoria Gray couldn’t be more pleased with its results. Calling her current condition “great,” she reports that all symptoms of her sickle cell anemia have disappeared completely since she underwent experimental CRISPR-driven treatments more than two years ago.

Gray participated in the very first study in the United States to apply CRISPR in an attempt to treat a genetic disorder. The positive effects of Gray’s pioneering treatment were both rapid and dramatic. The pain and fatigue that had plagued her throughout her life were suddenly gone and she felt as if she could finally take proper care of herself and her family. Even better, she no longer had to suffer the terrible side effects of her strong pain medication or rush to the emergency room on a regular basis to get life-saving blood transfusions.

After seeing dramatic improvements almost immediately after Gray left the hospital, the study team at the Sarah Cannon Research Institute were absolutely overjoyed with the results. However, the question remained: would these positive changes last?

Gray’s condition, more than two years since her CRISPR gene editing treatment, indicates that its effects are, indeed, long-lasting if not permanent. In the words of study head Dr. Haydar Frangoul, CRISPR has the proven ability to change patients’ lives without the “horrible complications” that often go hand in hand with existing treatments.

In fact, Gray has done so well over the past two years, that she is no longer an official subject in the groundbreaking study for which she volunteered several years ago. Upon qualifying for the study, physicians harvested marrow cells from her bones for laboratory editing using the CRISPR technique. After making exacting and targeted changes to the DNA of these blood-producing cells, researchers reintroduced billions of these modified cells into Gray’s body, where they immediately began to address the symptoms of sickle cell by producing a natural protein called fetal hemoglobin.

The dramatic health improvements engendered by this process were not exclusive to Victoria Gray. Of the 45 sickle cell patients that medical clinicians have now treated using CRISPR gene editing, at least 22 have exhibited significant gains according to the latest reported data.

And for her part, Gray can scarcely believe her good fortune and her entire family has expressed tremendous gratitude. Speaking with NPR from her home in Forest, Mississippi, she describes herself as being entirely symptom-free and “much more” than fine.

For their part, Dr. Haydar Frangoul and the rest of the Sarah Cannon Research Institute team are extremely excited about the vast potential of this rapidly evolving treatment method. They welcome the news that Boston’s Vertex Pharmaceuticals, in partnership with Cambridge’s CRISPR Therapeutics, plans to seek FDA approval for the CRISPR gene editing technique within the next one-and-a-half to two years.

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