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Boston-Based Vertex Aims to Tackle Sickle Cell Disease, Inspire Students

Researchers at Vertex Pharmaceuticals are hoping to use gene editing to restore blood cells impacted by sickle cell disease, which disproportionately affects Black people

NBC Universal, Inc.

Researchers at a Boston pharmaceutical company hope to make sickle cell disease — a blood disorder that disproportionately affects Black people — more manageable for patients.

Vertex Pharmaceuticals says it is trying to use gene editing to restore impacted blood cells and reduce symptoms. And the scientists working on the solution hope their actions will help inspire a new generation of young thinkers.

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"Growing up in a community of color, it was something that was present," Crew Smith, director at Vertex, told NBC10 Boston. "Not always in your face, but you knew people who had it. And also I knew people in my family, relatives who have the disease."

Finding transformative change for people who live with the condition is Smith's daily mission. The scientist from St. Thomas, with degrees from Princeton and Yale, works with colleagues to attack the underlying causes of the disease that's most commonly found in Black people.

"It's amazing to be able to be part of the process to be able to bring the idea of hope," she said.

Hope is also what Vertex offers Boston Public School students through its STEAM program.

"When they walk out, there's a twinkle in their eye," said Melodie Knowlton, a Harvard graduate and the company's global head of STEAM education. "They generally love the experience."

The program provides field trips for students to Vertex's learning lab.

"In here, we try to make it that this is about discovery, this is about what's coming, it's about how do you think about a problem, how do you approach it, versus a set of facts that you have to try to memorize," she said.

Students leave the lab enlightened. For Knowles, the work is personal.

"To be able to take my own personal passion for science and for STEM and what it has done for me in my life, and opened up possibilities and opportunities, and to help other brown and Black children see that for themselves," she explained.

"I come to work every day because I know that we are trying to make others' lives better," Smith said. "To be in that mission and on that road with other scientists of color is also an inspiration, and knowing that what we are doing is also paving the way for many others to come and impact this field of research, science and medicine."

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