Cure Sanfilippo Funds CRISPR Gene Editing of Neural Stem Cells

November 28, 2018
Team conducting CRISPIR gene editing for Sanfilippo Syndrome, MPSIII

Cure Sanfilippo Foundation is excited to grant funding to LA BioMed for the advancement of Dr. Michelina Iacovino’s work in the field of regenerative medicine for the treatment of Sanfilippo Syndrome (MPS III).

This innovative work will utilize CRISPR gene editing to transform patients’ own iPSC derived neural stem cells to supply critical enzyme and regenerative factors to the brain.

Dr. Iacovino’s, Assistant Professor of Pediatrics at LA Biomed/Harbor UCLA, research career has focused on the use of stem cells in multiple disorders, MPS diseases, and blood coagulation. Her ongoing work in parallel to this research grant will further develop the iPSC approach to MPSIIIB in her lab.

Dr. Iacovino commented: “We believe that this project will provide initial data essential for the development of a Neural Stem Cell therapy to treat Sanfilippo syndromes.”

The application of NSCs in neurodegenerative disorders, such as Sanfilippo syndrome, is particularly exciting because they could address both the underlying enzyme deficiency of Sanfilippo syndrome, while also secreting special substances that help support and promote healthy brain tissue.

This fascinating process transforms patients’ own skin cells back into the most basic stem cells in the human body.

These newly-formed primitive stem cells are then directed in the lab to become stem cells specialized to the brain (neural stem cells).

Neural stem cells (NSCs) are the cells that give rise to neurons and supporting cells(glia) of the central nervous system. NSCs also supply the brain with factors like brain derived neurotrophic factor to keep it healthy. CRISPR gene modification of the NSCs will then allow the cells to produce enough MPSIIIA enzyme to supply what is missing in the brain.

“Using a patient’s own cells and modifying them as needed, drastically reduces concerns about the body having a reaction to the cells, therefore allowing the regenerative cells to remain in the brain and provide benefit indefinitely. This would be an incredible achievement and benefit to the children suffering from MPSIII,” says Dr. Cara O’Neill, Chief Scientific Officer at Cure Sanfilippo Foundation.

Link to Michelina Iacovino PhD:

Link to article on MPSIIIB iPSCs:

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