engleski

The rat model for ALS and its potential in development of stem cell therapy

Amyotrophic lateral sclerosis (ALS) is a fatal and still incurable neurodegenerative disease characterized by fast deterioration of motor neurons. Death caused by paralysis occurs only 3 to 4 years after the first symptom is observed. Although majority of the cases are sporadic, familial subtype of ALS characterized by dominant inheritance attracted attention for mutation in superoxide dismutase 1 (SOD1) gene. In order to get insight in the pathological background of ALS, corresponding rat and mouse model encompassing various modifications of SOD1 gene are constructed. Moreover, availability of SOD1 animal models offers possibility to test various therapeutic approaches, including cell therapy and drug screening. In order to test potential of neural stem cell therapy in the rat model of ALS, in vitro differentiation of neural stem cells obtained from embryo cortex of SOD1 mutant rat was analyzed. Observed facts included shift in expression of various differentiation markers and disturbed cell clustering. In order to test potential beneficial effect using blood delivery approach, 10 millions of wild type nestin/GFP positive cells were injected in the rat tail vein. Interestingly, after 3 days, injected cells were found in the cortex and hippocampus of treated animals. Various ALS animal experimental models enable to test innovative therapeutic approaches for this fatal disease. Presence of injected neural stem cells in different regions of ALS affected rat brain after blood stream delivery represents important step further in cell transplantation therapy.

ALS; neurodegeneration; stem cells

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