Risk Linked to Stem Cell Transplantation can be Reduced by Gene Therapy

Genetically modifying stem cells prior to transplantation can help minimize the risk associated with the therapy, say researchers from New York-Presbyterian Hospital/Weill Cornell Medical Centre.

Stem cells intended to treat or cure a disease can end up wreaking havoc simply because they are no longer under the control of the clinician.

"Stem cell therapy offers enormous potential to treat and even cure serious diseases. But wayward stem cells can turn into a runaway train without a conductor," said senior author Dr. Ronald G. Crystal, chief of the Division of Pulmonary and Critical Care Medicine at NewYork-Presbyterian Hospital/Weill Cornell Medical Center.

"This is an issue that can be dealt with and we have the technology to do that in the form of gene therapy," he added.

Researchers said that one of the biggest potential problems with stem cell therapy is the development of tumors.

Also stem cells directed to become beating heart cells might mistakenly end up in the brain. Or insulin-producing beta cells which can't stop means the body can no longer regulate insulin levels.

The best way to avoid the problems is genetic modification of the stem cells prior to actually transplanting them, Dr. Crystal said.

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"Almost all therapeutics we use have a half life. They only last a certain amount of time," he said.

"Stem cells are the opposite. Once the future stem cell therapist does the therapy, stem cells have the innate potential to produce more cells," he added.

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The study appears in the journal Cell Stem Cell.

Source-ANI
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