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New gene therapy eliminates need for bone marrow transplant. Here's how it works.

Long Island boy undergoes groundbreaking gene therapy
Long Island boy undergoes groundbreaking gene therapy 02:17

NEW YORK -- A young Long Island boy is the first in New York state to undergo a new gene therapy for a rare blood disease.

A gene was actually added to the child's blood cells to cure a life-threatening ailment.

Just after he turned 8, Yusuf Saeed celebrated another birthday of sorts -- a rebirth of his health.

"We're super fortunate that we were able to get Yusuf the gene therapy so early on," mother Yusara Ahmed said.

The Valley Stream second grader was born with beta thalessemia, a genetic blood disorder. Unable to make red blood cells, Yusuf required transfusions every few weeks.

A bone marrow transplant could have cured him, but there were no matches, not even his twin sister.

"The time passing and we were not finding any donor," father Farrukh Saeed said.

As hope ran out for a donor, a novel new gene therapy was approved by the Food and Drug Administration. A first in New York, a gene was added to Yusuf's stem cells.

"Gene therapy is a transformative treatment. The patient is his own donor. We actually collect the patient's own stem cells," said Dr. Banu Aygun, a hematologist at Cohen Children's Medical Center.

The altered cells are then infused back into the patient, a one time treatment that prompts bone marrow to do its job.

"He was not needing a blood transfusion for the first time in his life, since mid February," Dr. Jon Fish said. "This is really exciting and transformative moment because this is just the beginning."

Gene therapy shows promise with similar diseases and eliminates the need for a bone marrow transplant, which Yusuf's aunt needed when she was a child.

"The only option during my sister's time was to go through a donor, and so now for that to be the only option is really, really amazing. It's mind blowing," Ahmed said.

Yusuf shared few words, but made them count.

"Thank you so much Dr. Aygun and Dr. Fish for taking care of me," he said.

His parents said they hope he will be a voice of hope.

"One person can help another. That's how community grows. So I would just hope he becomes that for other kids," Ahmed said.

The hope of doctors is that Yusuf can now live a long, healthy life without ever needing another blood transfusion.

The first patients who underwent this gene therapy in clinical trials are already eight years out, living transfusion-free.   

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