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Updated: Aug 3, 2026

Experimental Strategies to Bridge Large Tissue Gaps in the Injured Spinal Cord after Acute and Chronic Lesion
Published on: April 5, 2016
Designing cell- and gene-based regeneration strategies to repair the injured spinal cord
1The Miami Project to Cure Paralysis, Department of Neurological Surgery, University of Miami Leonard M. Miller School of Medicine, Miami, Florida 33101, USA. DPearse@miamiproject.med.miami.edu
Abstract:
There is an array of new and promising strategies being developed to improve function after spinal cord injury (SCI). The targeting of a diversity of deleterious processes within the tissue after SCI will necessitate a multi-factorial intervention, such as the combination of cell- and gene-based approaches. To ensure proper development and design of these experiments, many issues need to be addressed. It is the purpose of this review to consider the strategies involved in testing the efficacy of these new combinations to improve axonal regeneration. For cell-based therapy, issues are choosing a SCI model, the time of cell implantation, placement of cells and their subsequent migration, fluid versus solid grafts, use of agents to prevent immune rejection, and tracking of implanted cells. Grafting is also discussed in view of improving function, reducing secondary damage, bridging the injured spinal cord, supporting axonal regrowth, replacing lost neurons, facilitating myelination, and promoting axonal growth from the implant into the cord. The choice of a gene delivery system, gene-based therapies in vivo to provide chemoattractant and guidance cues, altering the intrinsic regenerative capacity of neurons, enhancing endogenous non-neuronal cell functions, and targeting the synthesis of growth inhibitory molecules are also discussed, as well as combining ex vivo gene and cell therapies.
Insights
Combining cell and gene therapies shows promise for improving function after spinal cord injury (SCI). This review examines strategies for testing these multi-factorial interventions to enhance axonal regeneration and recovery.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Biotechnology
Background:
- Spinal cord injury (SCI) presents complex challenges to neural tissue repair.
- Current research focuses on multi-factorial interventions to address diverse pathological processes post-SCI.
- Combining cell- and gene-based therapies is a promising strategy for functional recovery.
Purpose of the Study:
- To review strategies for testing the efficacy of combined cell- and gene-based therapies for improving axonal regeneration after SCI.
- To identify key considerations for designing preclinical studies involving these complex interventions.
Main Methods:
- Discussion of critical factors for cell-based therapies, including SCI model selection, cell implantation timing and placement, graft type, immune rejection prevention, and cell tracking.
- Exploration of gene delivery systems and in vivo gene-based therapies for providing chemoattractant and guidance cues.
- Consideration of strategies to enhance intrinsic neuronal regenerative capacity and endogenous non-neuronal cell functions.
- Discussion of targeting growth-inhibitory molecules and combining ex vivo gene and cell therapies.
Main Results:
- The review highlights numerous challenges and considerations for successful cell-based transplantation, such as graft integration and immune response.
- It outlines various gene therapy approaches, including those aimed at promoting axonal growth and neuronal survival.
- The synergistic potential of combining cell and gene therapies for comprehensive spinal cord repair is emphasized.
Conclusions:
- Optimizing cell- and gene-based strategies requires careful consideration of numerous experimental variables.
- Multi-factorial interventions, particularly combined cell and gene therapies, hold significant potential for advancing spinal cord injury treatment.
- Further research is needed to refine these approaches and translate them into clinical applications.
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