Targeting Telomerase for Cell Therapy
PATENT STATUS
A PCT application disclosing this invention was filed in April 2016.
OVERVIEW OF TECHNOLOGY ON OFFER
A large population of stem cells with the appropriate telomerase lengths for safe in vivo cell therapy and the method to engineer the said stem cells.
BRIEF DESCRIPTION
The propensity of stem cell regenerative therapies to lead to teratoma formation in patients is a major obstacle to the acceptance of stem cell therapies in the clinic and can be attributed to the increased telomerase activity in stem cells. Shortening of telomeres by reducing telomerase activity is a logical step but telomeres that are too short can lead to genomic instability. In 2015, Assistant Professor Li Shang, a Principal Investigator in the Cancer and Stem Cell Biology Programme at Duke-NUS, and co-workers devised a method to create a large population of safe stem cells by knocking out telomerase via CRISPR-Cas or the Cre-Lox recombinase system. Crucially, the stem cells were also subjected to a transient overexpression of telomerase such that the combined effect of knockout and overexpression leads to a population of stem cells which are further passaged and selected for telomere lengths of approximately 5kb. This length dramatically reduces the predisposition of stem cells to form teratomas in vivo, a safety feature essential to a wider acceptance of cell therapies in clinic. These stem cells with shortened telomeres could also be differentiated into cells of different lineages such as neurons, astrocytes, glia and haematopoietic cells, highlighting that telomere lengths and the function of stem cells to generate the necessary cell type in a patient are disconnected features. The large population of “safe” stem cells and the method to generate them is a promising technology that can find wider acceptance in the stem cell therapy industry.
POTENTIAL APPLICATIONS
Stem cell therapy
KEY BENEFIT
Generating a large population of “safe” stem cells displaying the appropriate telomere lengths for therapy.
INVENTOR BIO
Li Shang
CONTACT
Please email us for further enquiries: cted@duke-nus.edu.sg