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 are your 2019 research resolutions?

Mass General Research Resolutions

MGH researchers explain their fields of study and share their New Years resolutions in this thoughtful piece featuring Drs. Fatima Cody Stanford, Nitya Jain, Shannon Stott, Helen Riess, Sareh Parangi, Pike See Cheah, Guisy Romano-Clarke, and our very own PI, Jonathan Hoggatt. This year, the Hoggatt lab hopes to better understand hematopoietic stem cell heterogeneity to enhance bone marrow transplantation.

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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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