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CMCC 2519

Reprogramming Committed Blood Cells Back to Hematopoietic Stem Cells

Inventors: Derrick Rossi, Jonah Riddell, Roi Gazit

Invention Types: Research Tool, Therapeutics

Research Areas: Regenerative Medicine, Stem Cell

Keywords: Stem/Progenitor Cell

For More Information Contact:  Dietz, Ryan

 

Invention Description:

Hematopoietic stem cells (HSC) are multipotent, self-renewing precursor cells capable of differentiating into all specialized blood and immune cells of the body. Since the late-20th century, hematopoietic stem cell transplantation (HSCT) has been used as a treatment for hematologic cancers, autoimmune diseases, and various genetic disorders. Though widely adopted for clinical use, there remain several factors that limit successful outcomes in HSCT procedures, including the number of HSCs transplanted, graft failure, infection, and graft versus host disease (GVHD). GVHD is associated with significant morbidity and mortality in nearly ~70% of allogeneic transplantations.


Researchers in the Rossi lab at Boston Children’s Hospital have discovered a combination of six transcription factors capable of imparting multi-lineage transplantation potential onto committed blood cells. Their work also identified two additional transcription factors that greatly improved reprogramming efficiency when administered in conjunction with the original combination of factors. The HSC generated through this process, termed induced HSC (iHSC), were demonstrated in a mouse model to be multipotent, self-renewing and serially transplantable.

Applications:

• Potential source of patient-specific HSCs for autologous transplantation

• Technology for the generation of autologous HSCs from committed blood cells

• Research tool to study the disease etiology of hematological disorders

• Cell-based platform for therapeutic screening

Competitive Advantages:

• Differentiated blood cells are far more abundant than natural HSCs (1/10,000 in bone marrow, 1/100,000 in peripheral blood)
• iHSC can be generated from autologous sources and should display lower levels of immunogenicity

Business Opportunity:

Non-exclusive license or sponsored research opportunity.

Key Publications: Ridell J, Gazit R, Garrison BS, Guo G, Saadatpour A, Mandal PK, Ebina W, Volchkov P, Yuan G-C, Orkin SH, Rossi DJ. Reprogramming Committed Murine Blood Cells to Induced Hematopoietic Stem Cells with Defined Factors. Cell. 2014. 157(3): 548-564.

IPStatus: Pat. Pend.