Young Girl’s Life Transformed by Breakthrough Gene Therapy

Quick Smiles:

• A young girl, Rahemeen Nabeel, is living pain-free after receiving a groundbreaking gene therapy for beta-thalassemia.
• The treatment, called Zynteglo, was developed at the Children’s Hospital of Philadelphia and uses the patient’s own cells.
• Dr. Timothy Olson and his team are hopeful as more patients are set to receive this transformative therapy.

In a remarkable medical breakthrough, a young girl named Rahemeen Nabeel is now living a life free from the debilitating symptoms of beta-thalassemia, a genetically-inherited blood disorder.

This achievement comes thanks to Zynteglo, a pioneering gene therapy developed at the Children’s Hospital of Philadelphia.

Zynteglo works by using the patient’s own cells to create stem cells, which are then reprogrammed to replace faulty genetic copies.

“To see a therapy like this move into being widely available for patients, this is why we do what we do,” shared Dr. Timothy Olson.

Rahemeen, who once struggled with significant pain and required regular blood transfusions, now finds herself with an abundance of energy.

Her mother, Zainab Nabeel, recounted their relentless search for treatment:

“So we took her to Paris, we took her to Italy, Rome, Switzerland, and then we took her to Turkey, Korea.”

Despite the challenges, including undergoing chemotherapy and losing her hair, Rahemeen’s journey has been a triumph.

“It was a long, stressful journey, but we are really, really happy,” expressed Zainab Nabeel.

Dr. Olson confidently states that Rahemeen is cured, and he is optimistic about the 17 other patients currently in various stages of treatment.

Before the advent of CRISPR and Zynteglo, the only cure available was a bone marrow transplant from a closely related donor, a difficult and often unattainable option for many.

Zynteglo and similar treatments utilize “Yamanaka factors” to convert normal cells into pluripotent stem cells, capable of becoming almost any cell type.

These factors are named after Shinya Yamanaka, the Nobel Prize-winning biologist who discovered them.

This journey of hope and healing is a testament to the power of medical innovation and the relentless pursuit of a better quality of life for patients like Rahemeen.

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