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04 May 2015
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Creating Neurons from Stem Cells: Possible Treatment for Neurodegenerative Disorders

At the University of California, San Francisco (UCSF), a research team has identified a long non coding RNA molecule, pnky which can be knocked down to increase the production of neurons in neural stem cells of humans. This discovery can serve as an important key in regenerative medicine that includes treatment for disorders such as cancer, traumatic brain injury, Alzheimer’s disease, Parkinson’s disease etc. The research work led by neurosurgeon Daniel A. Lim and team was published in the journal, Cell Stem Cell.

About fifty thousand long non coding RNA (lncRNA) molecules were newly discovered in the human genome and pnky is one among them. Although, some of these RNA molecules do not code for proteins, they seem to have other biological functions. The pnky molecule stretches to 200 or more nucleotides in the human genome and expressed in the neural stem cells. It is observed that pnky is found only in the brain and the co-authors first studied it in embryonic mouse cortex and later identified in neural stem cells of developing human brain. Pnky, along with its splicing regulator, PTBP1 regulates neurogenesis (formation of neurons) and alternative splicing in neural stem cells.

When pnky is removed from the neural stem cells by the process of ‘knocking down’, the team observed an increase in the neuron production by multiple folds. Dr. Lim and team believe that the increased neuron production can find applications in cancer therapy and regenerative medicine. Using mass spectrometry, the team found pnky binding to the splicing regulatory protein, PTBP1 and their link to brain tumor. According to the study, pnky and PTBP1 causes the brain tumor growth; removing either one of them can suppress the production of neurons and thereby the tumor growth. Lim said, “It is possible that pnky can regulate the growth of brain tumor, which means we may have identified the target for the treatment of brain tumor.”

Based on this study, it is evident that the knockdown of lncRNA molecule, pnky causes increased neuron production or neurogenesis. Thus, it helps in developing better treatment options for various disorders and neurodegenerative diseases in humans.

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