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CNS Rescue and Repair

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Overview

A primary research theme in our lab is the development of novel approaches aimed at nervous system repair, regeneration and recovery. An especially promising area is cell-based transplantation therapies, whereby neural stem cells can be used to generate specific cell types in order to replace those cells compromised by disease or injury. In addition, somatic stem cells can be engineered as delivery vehicles for therapeutic factors for neuroprotection and regeneration. Coupling these approaches with biocompatible nano/micro materials is an especially powerful tactic for development and implementation of experimental strategies for neural tissue engineering and therapeutics.

Stem Cell Transplants for Treatment of Neurodegenerative Conditions

As a potential therapy for treatment of neurodegenerative diseases, stem cells and neural progenitor cells (NPCs) have been proposed as unique sources of transplantable cells to provide neuroprotection to the remaining CNS elements and to replace degenerating neurons and glia. We are using this strategy and are evaluating the survival, differentiation and integration of NPCs and stem cells transplanted into normal and damaged retinas in animal models used to study neurodegenerative conditions.

Furthermore, we have genetically engineered MSCs to produce and secrete bioactive neurotrophic factors for delivery to the injured CNS.

The MSCs may be used for autologous transplantation and therefore minimize the likelihood of immune rejection of the grafted cells. Engineering stem cells ex vivo for neural repair strategies may ultimately provide a reliable means of long-term delivery of therapuetic factors.

Image-based High Content Screening (HCS)

An important step in implementing cell-based delivery of neuroprotective compounds is to determine the health of the engineered cells. Employing cellular image-based high content screening is a powerful approach to rapidly assess multiple cell parameters. Promising results have been obtained by cellular reprogramming and by genetic modification of cells to secrete neurotrophic factors; combining biomaterials to develop bio-mimetic strategies can lead to further advancement in regenerative therapies.