Stem cells are unique because they undergo self-renewal. When a stem cell divides it can split into two stem cells, a stem cell and a more differentiated cell or two differentiated cells. Dr. Dirk Loeffler is studying how a stem cell knows what cell type to become and the factors that influence this stem cell identity.
Stem cells are influenced by the environment in which they grow. At St. Jude Dr. Marta Derecka is studying the interplay between stem cells and the bone marrow microenvironment, looking at the transcription factors that regulate how the environment functions and, in disease, can contribute to treatment resistance or immune suppression.
A class of conditions called bone marrow failure syndromes can be a precursor to cancer if not properly diagnosed. Dr. Marcin Wlodarski is an expert in bone marrow failure. His laboratory studies the genetics of these disorders and defined the contributions of SAMD9/SAMD9L mutations to bone marrow failure.
Find out more: Tailoring treatment for bone marrow failure syndromes.
When a disease is caused by a single genetic mutation, it is possible to fix the mutation and cure the disease using gene editing. At St. Jude, scientists led by Dr. Mitch Weiss, have studied multiple gene editing approaches to treat and cure sickle cell disease, including Cas9 nucleases, base editing and prime editing.
Find out more: Gene editing holds the promise of a sickle cell cure.
St. Jude has launched a new initiative, the partnership to advance development of individualized genomic medicines, or PARADIGM. Through PARADIGM we will directly address blood disorders caused by rare genetic mutations to create bespoke gene therapies.