Gene Therapy Helps Patients Diagnosed with X-Linked Adrenoleukodystrophy

Boston (DbTechNo) – Researchers are reporting a new breakthrough in the ongoing battle against a brain disease called X-linked adrenoleukodystrophy using gene therapy.

The brain condition is hereditary and is characterized by the slow wearing down of fatty acids which are meant to protect nerves in the brain.

Over time as the condition worsens, the patient becomes more physically and mentally challenged until they eventually die.

The condition only effects males, and up until now was only treated by administering bone marrow transplants.

Two young boys from France diagnosed with the rare condition received a combination of gene therapy and stem cells, resulting in the slowing down and eventual stopping of the deterioration of protective fatty acids.

“This is the first time that a very severe brain disease has been treated with efficacy by gene therapy,” said lead researcher Patrick Aubourg, a professor of paediatrics at University Paris-Descartes.

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