Omarion, a former SCID patient who was cured using gene therapy.

One-Shot “Miracle Cures” That Can Cost Millions

Gene therapies have been incredibly successful for many rare diseases, especially diseases of the blood and immune systems. For example, patients with severe combined immunodeficiency syndrome (SCID), commonly known as bubble boy disease, can be cured after receiving a bone marrow transplant using their own cells that have been genetically altered to add back the missing gene whose absence leads to the disease. However, patients, drug companies, and governments alike must grapple with the fact that these “miracle cures” are often exorbitantly priced.

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Gene editing therapy for bubble boy disease has a money back guarantee

ADA-SCID, commonly known as bubble boy disease, is a immune system disease caused by a mutation in the adenosine deaminase gene. It is commonly treated by bone marrow transplantion from a donor. Now, pharmaceutical company GlaxoSmithKline has developed an new gene editing therapy called Strimvelis, which uses retroviral gene editing to insert a healthy gene into a patient’s own cells, with a money back guarantee if it doesn’t work. Read more about the implications of this in a CNN article by Susan Scutti, featuring commentary by our PI, Dr. Hoggatt:

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What defines a stem cell?

What Defines a Stem Cell? Scientists Rethink the Answer

After several puzzling discoveries have proven that certain stem cell populations are not stem cells at all, or that some differentiated adult cells are able to revert to a stem-like state in certain situations, scientists are redefining what it means to be a stem cell. Dr. Hans Clevers, Dr. Jonathan Hoggatt, and Dr. Pamela Robey discuss tissue repair, phony treatments, and the controversy of what characterizes “stemness” in this piece by Jordana Cepelewicz of Quanta Magazine.

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