Enhanced hematopoietic stem cell regeneration

Quick Facts:
Tags: Bone marrow, Fatty acid, Gene therapy, Genome editing, Haematopoiesis, Hematopoietic stem cell, Hematopoietic stem cell transplantation, Metabolism, Peroxisome, Progenitor cell, Protein, Stem cell, Stem-cell therapy
Inventors: Lei Ding, Yue Zhang
Manager: Kristin Neuman
Departments: Microbiology, Rehabilitation & Regenerative Medicine
Divisions: Columbia University Medical Center (CUMC)
Reference Number: CU25234
Release Date: 2026-02-26

This technology is a method for stimulating hematopoietic stem cell (HSC) and progenitor cell regeneration to improve bone marrow transplants and other regenerative therapies.

Unmet Need: Improved stem cell regeneration for transplants and other regenerative therapies

Hematopoietic stem cells (HSCs) and progenitors are cells that regenerate the blood and immune systems and are used for their self-renewal properties to replenish the hematopoietic system after injury or during transplantation. HSCs used for serial transplantation gradually lose their self-renewal and blood cell output with each round of transplantation. Currently, there are no methods to regulate or control the efficiency of self-renewal in HSCs and progenitors.

The Technology: Partial inhibition of fatty acid metabolism enhances hematopoietic stem cell regeneration

This technology identifies the unique role that peroxisomes, metabolic organelles that break down long-chain fatty acids, play in hematopoietic stem cell (HSC) regulation. Inhibition of this metabolic process results in extensive self-renewal in HSCs and progenitors. Deleting a key protein in this pathway results in greater multilineage reconstitution and a higher contribution of HSCs and progenitors in mice. By controlling this pathway, this technology has the potential to augment the effectiveness of HSCs in bone marrow transplantation and other regenerative therapies.

This technology has been validated with mouse models with no adverse effects.

Applications:

  • Stem cell therapy for regenerative therapies
  • Improvements to bone marrow transplantations
  • Research tool for studying stem cell regeneration
  • Research tool for studying multi-lineage differentiation
  • Efficient generation of gene-edited stem cells for gene therapy

Advantages:

  • Enhances the efficacy of existing therapies
  • Expands hematopoietic stem cells and progenitors efficiently
  • Presents no adverse effects in in vivo models

Lead Inventor:

Lei Ding, Ph.D.

Patent Information:

Patent Pending

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