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Cell-based Therapy for Neurological Disorders

WIB-2952

APPLICATIONS OF TECHNOLOGY:

ADVANTAGES:

ABSTRACT:

Axel Visel of Berkeley Lab, John Rubenstein of UC San Francisco, and their team of researchers have discovered 145 gene enhancers – short pieces of DNA that act as molecular switches – that turn on gene expression in specific parts of the brain. The inventors are the first to map exactly where these 145 enhancers drive expression in the forebrains of transgenic mice. This unique catalog will enable biomedical researchers to develop more targeted, cell-based treatments (including stem cell and regenerative therapies) that replace dying brain cells through cell transplants, and may be more effective than drugs at treating neurological and psychiatric disorders.

The Berkeley Lab/UCSF team has also invented a method for growing specific types of neurons or brain cells for cell replacement therapies or cell-based assays to produce from stem cells the specific neuronal cell types needed by a patient. It can also be used to verify that the generated cells have the desired properties and are ready for use in transplants or cell-based assays.  Finally, the method can also be used to produce many different brain cell types for the screening of beneficial or toxic effects of drugs in test tube experiments, instead of testing them on animals or humans. 

Therapies that replace dying neurons through cell transplants offer a promising alternative to conventional drug treatments.  However, researchers are currently limited in their ability to make the correct types of neurons required for such applications, owing to the technical difficulties of generating the desired cellular phenotypes and characterizing their properties. The Berkeley Lab/UCSF invention addresses this critical bottleneck in available treatments for patients who use drug-based therapies yet still experience disabling symptoms.

DEVELOPMENT STAGE:  Bench-scale prototype

STATUS: Patent pending.  Available for licensing or collaborative research.

FOR MORE INFORMATION:

Visel, A., Blow, M. Li, Z., Zhang, T., Akiyama, J., Holt, A., Plajzer-Frick, I., Shoukry, M., Wright, C., Chen, F., Afzal, V., Ren, B., Rubin, E., and Pennachhio, L. “ChIP-seq accurately predicts tissue-specific activity of enhancers,” Nature 457 (2009), 854-858.

SEE THESE OTHER BERKELEY LAB TECHNOLOGIES IN THIS FIELD:

Potential Prognostic Markers and Therapeutic Targets for Neurological Disorders, IB-2601

REFERENCE NUMBER: WIB-2952

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Last updated: 09/11/2012