AREAS OF RESEARCH

Understanding and designing blood

Our lab aims to understand how blood develops, with the goal of informing novel strategies for cell engineering and therapy.

Simplified blood hierarchy figure
Blood disorders icon

CRISPR cures for blood disorders

HSCs are multipotent stem cells that give rise to all cells of the blood system. Gene correction at HSC level can ameliorate many diseases such as sickle cell disease, thalassemia, leukemia, lymphoma and primary immunodeficiency. We aim to develop CRISPR-based experimental therapeutics against blood disorders and deliver these therapies to patients. To this end, we are advancing next-generation delivery platforms to edit HSCs.

Blood disorders icon

CRISPR cures for blood disorders

HSCs are multipotent stem cells that give rise to all cells of the blood system. Gene correction at HSC level can ameliorate many diseases such as sickle cell disease, thalassemia, leukemia, lymphoma and primary immunodeficiency. We aim to develop CRISPR-based experimental therapeutics against blood disorders and deliver these therapies to patients. To this end, we are advancing next-generation delivery platforms to edit HSCs.

Simplified blood hierarchy figure
Gene lineage differentiation figure
T cell fate icon

Genetic and Epigenetic control of T cell fate

T lymphopoiesis is a complex developmental process that takes place in the thymus, which generates a diverse repertoire of cytotoxic (CD8+), helper (CD4+) and regulatory (CD4+FOXP3+) T cells that mediate adaptive immune responses. We use functional genomics to uncover the genetic and epigenetic determinants that regulate each T cell subset. Elucidation of the networks that govern T cell fate in health and disease will guide our efforts to develop novel T cell therapies.

Engineering immune cells icon

Engineering immune cells from iPSCs

Recent advances in adoptive T cell therapies (ACT) have revolutionized cancer immunotherapy and autoimmune disease treatment. However, an important step towards the broad applicability of ACT is an “off-the-shelf” T cell product. Human iPSCs represent a potentially unlimited source of T cells for ACT. We recently established a novel platform to generate large numbers of T cell progenitors from iPSCs (Vo et al. Nature, 2018).  We aim to develop this system toward two goals: (1) engineering antigen-specific T cells for cancer immunotherapy and (2) generating regulatory T cells  (Tregs), master controllers of an overactive immune response,  to prevent and treat autoimmune disease and organ transplant rejection.

Engineering immune cells figure
Engineering immune cells figure
Engineering immune cells icon

Engineering immune cells from iPSCs

Recent advances in adoptive T cell therapies (ACT) have revolutionized cancer immunotherapy and autoimmune disease treatment. However, an important step towards the broad applicability of ACT is an “off-the-shelf” T cell product. Human iPSCs represent a potentially unlimited source of T cells for ACT. We recently established a novel platform to generate large numbers of T cell progenitors from iPSCs (Vo et al. Nature, 2018).  We aim to develop this system toward two goals: (1) engineering antigen-specific T cells for cancer immunotherapy and (2) generating regulatory T cells  (Tregs), master controllers of an overactive immune response,  to prevent and treat autoimmune disease and organ transplant rejection.