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Regenerative gene therapy


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Spinal cord injuries frequently result in the irreversible loss or damage of neurons. Therefore, much research is focusing on the engrafting of external cells to replace the loss. Of course, this comes with a different set of challenges. A research team led by Prof. Chen (Pennsylvania State University, USA) found an entirely different approach to this difficult problem.

Gene therapy to recruit glial cells
Prof. Chen and his team used a retrovirus to force a special type of glial cells, astrocytes, to express a gene that transforms them into neurons. Spinal neurons could be generated only by using spinal glia. This greatly increases the potential of this approach, as it means that the gene therapy should simply target the spinal cord.

Efficacy at different timepoints
Probably the most interesting aspect of this technique is the timing of its application. Tests on experimental models showed that this neuronal conversion could be achieved on two time-points: ten days and four months after spinal cord injury. In both cases the newly generated neurons were functional and could integrate within the already existing spinal circuitry.

Next steps towards the patient
These studies provide the proof that in principle, astrocyte-to-neuron conversion can be used to regenerate functional new neurons. Yet, further testing is required before the technique could be potentially developed into therapeutic interventions for humans.

This study was published in the journal Frontiers in Cell and Developmental Biology.

Source: “Regeneration of Functional Neurons After Spinal Cord Injury via in situ NeuroD1-Mediated Astrocyte-to-Neuron Conversion.” by Puls B, Ding Y, Zhang F, Pan M, Lei Z, Pei Z, Jiang M, Bai Y, Forsyth C, Metzger M, Rana T, Zhang L, Ding X, Keefe M, Cai A, Redilla A, Lai M, He K, Li H, Chen G. Published in the journal Frontiers in Cell and Developmental Biology.

You can find out more on the spinal cord and the influence of its damage on the human body in our basic-information. Wings for Life Glossary gives an understanding of scientific terms.