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.
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:
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.
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.
Congratulations to Dr. Jonathan Hoggatt, who has received a 2019 ASH Scholar Award in the category of basic/translational research, one of the organization’s most prestigious awards!
Our principal investigator, Jonathan Hoggatt, discusses his passion for cooking in this ASH Clinical News interview, including his very own recipe for filet mignon meatballs!
To hear Dr. Hoggatt discuss his early experiments with chocolate chip cookies, click on the link below:
A Spanish language interview with Jon discussing new advances in gene therapy.
Jon discusses gun violence in America, and the impact on physicians and scientists.
Hear about our work to make donating bone marrow stem cells easier for the donor, and making the transplant better for the patient.