Trans-Differentiation Of Fibroblasts Into Functional Neurons
Trans-Differentiation Of Fibroblasts Into Functional Neurons
A novel strategy to engineering non-neural cells into functional neurons. Their strategy does not rely on the ectopic expression of lineage specific transcription factors, but on the modulation of a key gene, capable of regulating these transcription fact
San Diego, CA, United States
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Background

Neurodegenerative diseases result from the gradual and progressive loss of neural cells and lead to nervous system dysfunction. In the United States alone, four million patients suffer from Alzheimer's disease (AD), one million from Parkinson's disease (PD), 350.000 from multiple sclerosis (MS), and 20.000 from amyotrophic lateral sclerosis (ALS). Worldwide, these incurable neurodegenerative diseases produce immeasurable societal strain as they afflict more than 20 million people. Stem cell transplantation has recently emerged as an attractive alternative option for fighting neurodegenerative diseases; the transplantation of embryonic-stem-cell-derived dopaminergic neurons has been shown to be efficient in restoring motor symptoms in conditions of dopamine deficiency. Recent studies have shown that fibroblasts can be trans-differentiated into functional neurons by ectopic expression of at least three neuronal lineage specific transcription factors. Neuronal differentiation is a well-studied paradigm as a consequence of transcription reprogramming. The induction of pluripotency or trans-differentiation of one cell type to another can be accomplished with cell lineage-specific transcription factors. Neuronal differentiation is subject to additional layers of control, such as regulated RNA processing.


Technology Description

Researchers at the University of California have developed a novel strategy to engineering non-neural cells into functional neurons. Their strategy does not rely on the ectopic expression of lineage specific transcription factors, but on the modulation of a key gene, capable of regulating these transcription factors. The inventors have demonstrated that regulated expression of such key gene is able to induce massive reprogramming at both the splicing and microRNA levels to drive the cell fate decision towards the neuronal lineage. The invention has the potential to engineer non-neuronal cells into neurons in the brain to treat with neurodegenerative diseases.


Applications

The invention provides a new strategy to convert non-neural cells to functional neurons and may provide an effect way to treat neurodegenerative diseases Patients-specific neurons could also be used to generate in vitro models of diseases, to study the pathogenesis of neurodegenerative diseases, and to create platforms for drug screening and discovery and for drug toxicity testing. The invention may have broad impact on regenerative medicine.


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