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  4. Novel innovations in cell and gene therapies for spinal cord injury

Novel innovations in cell and gene therapies for spinal cord injury

F1000Research, 2020 · DOI: 10.12688/f1000research.21989.1 · Published: April 22, 2020

Spinal Cord InjuryRegenerative MedicineGenetics

Simple Explanation

Spinal cord injury (SCI) leads to chronic and multifaceted disability, which severely impacts the physical and mental health as well as the socio-economic status of affected individuals. Inhibitory factors present in the SCI microenvironment and the poor intrinsic regenerative capacity of adult spinal cord neurons are obstacles for regeneration and functional recovery. In this review, we highlight several potent cell-based approaches and genetic manipulation strategies (gene therapy) that are being investigated to reconstruct damaged or lost spinal neural circuits and explore emerging novel combinatorial approaches for enhancing recovery from SCI.

Study Duration
Not specified
Participants
Not specified
Evidence Level
Review

Key Findings

  • 1
    Gene therapy has gained promising advancement in the past decade, as six therapies have gained clinical approval for conditions such as spinal muscular atrophy or Leber’s congenital amaurosis.
  • 2
    Human iPSC-derived cells would instead allow for the potential generation of autologous cell lines whilst avoiding ethical issues.
  • 3
    Combinatorial strategies can greatly enhance the implementation and functional outcomes of these regenerative treatments.

Research Summary

Spinal cord injury (SCI) leads to chronic and multifaceted disability, which severely impacts the physical and mental health as well as the socio-economic status of affected individuals. Considerable progress has been made in recent years in developing cell and molecular approaches to enable the regeneration of damaged spinal cord tissue. Recent innovations in effective gene- and cell-based therapies have vastly improved the technical ability to induce regeneration in the spinal cord.

Practical Implications

Advancements in Gene Therapy

Gene therapy is advancing with clinical approvals for conditions like spinal muscular atrophy, offering potential for SCI treatment by enhancing pro-regenerative factors or suppressing inhibitory molecules.

Promise of Cellular Therapies

Cellular therapies using neural stem/progenitor cells (NSPCs) hold promise for SCI by replacing lost cells, modulating the immune system, and secreting neurotrophic factors. Human iPSC-derived cells offer a clinically attractive approach.

Combinatorial Treatment Strategies

Combining gene and cell therapies with rehabilitation training, neuromodulation, and biomaterials shows potential for enhancing regenerative treatments and improving functional outcomes in SCI patients.

Study Limitations

  • 1
    Ethical, technical, and safety concerns with allogeneic ES-derived cells
  • 2
    Suboptimal differentiation of transplanted NPCs in the injured cord microenvironment to non-neuronal cells
  • 3
    Limited integration of transplanted cells after SCI due to the graft’s discordant regional identity

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