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:
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!
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.