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Updated: Aug 30, 2026

Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
Published on: May 16, 2025
[A neurologist's views on cellular and gene therapy in nervous system diseases]
1Neurologická klinika 2. LF UK a FNM, Praha. martin.bojar@lfmotol.cuni.cz
Abstract:
Lesional impairment of the brain and spinal cord is a serious and usually irreversible disorder. There is only a small chance of functionally significant compensation of lesions. Neurons and glia are marked by the minimal regeneration. Though plasticity and adaptability represent important mechanisms, which can contribute to the compensation of structural and functional CNS disorders, irreversible structural and functional deficits may develop. Transplantation of various cells incl. neurons from fetal brain or transplantation of genetically modified cells was therefore proposed. Clear verification of this method by animal tests and by clinically controlled trials is a necessary condition. Its application is expected in atrophic-degenerative and vascular diseases of CNS, tumors, injuries, seizure, inflammatory and demyelinating diseases in which the replacement of neurons, glia and enhancement of their regeneration play the key role. Transplantation of embryonic stem cells, fetal neural cells, and haematopoietic stem cells introduced by stereotaxic, hematogenic or intrathecal procedure gets the attention. Bioethical problems and the possible contribution of this method based on experience with transplantation of fetal cells into basal ganglia of 3 patients with Parkinson's disease and haematopoietic stem cell transplantation in multiple sclerosis (n 1) are discussed.
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