Closing the gap to cure Fabry disease

Fabry  Philip S. Orsino Facility for Cell Therapy (University Health Network)

Supported by the Philip S. Orsino Facility for Cell Therapy (University Health Network) in Toronto and led by Dr. Armand Keating in collaboration with physicians across Canada, the world’s first gene therapy clinical trial for Fabry disease will soon launch. This rare inherited enzyme deficiency can shorten lifespan by as much as 40 years.

Researchers will remove a quantity of stem cells from a Fabry patient’s blood. Then a working copy of a new gene will be inserted into the stem cells using a specially engineered virus. During the final phase of the trial, researchers hope to transplant these stem cells back into the donor patient and the new, working copy of the gene will create the missing enzyme.

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