Innovation

RNAa For The Treatment Of Diabetes

University of California System: University of California, San Francisco
posted on 05/04/2010

Overexpression of specialized factors involved in Beta cell differentiation can differentiate or trans-differentiate non-Beta cells into insulin-secreting cells.  UCSF scientists have developed a technique utilizing small duplex RNAs to activate genes involved in Beta cell differentiation. 

Suggested Uses

  • Differentiated of cells by this method may offer a long-term solution to type 1 diabetes.

Advantages

  • Re-activation of endogenous genes involved in beta cell differentiation.
  • Does not require exogenous sources of DNA.
  • Circumvents concerns associated with viral vectors.

Innovation Details
 

Detailed Description

Type 1 Diabetes is a major health concern in the United States and other developing countries.  It occurs when the pancreas no longer produces insulin in response to increased blood glucose levels. Without frequent insulin treatment, people afflicted with type 1 diabetes become hyperglycemic, which can lead to major health problems. Beta cells are the source of insulin production in the pancreas. UCSF scientists have developed a technique utilizing small duplex RNAs to activate the expression of endogenous genes involved in Beta cell differentiation. Activation of such genes can lead to the differentiation or trans-differentiation of non-beta cells (i.e. embryonic stem cells, induced pluripotent stem cells, etc.) into insulin-producing beta cells. Currently, all methods of beta cell differentiation require exogenous sources of DNA (i.e. viral vectors, etc.) for gene overexpression. This technique offers a solution to conventional vector-based systems by circumventing the need to transduce/transfect cells with exogenous DNA sequence. Differentiation or trans-differentiation of donor cells into new beta cells can result in a permanent solution to type 1 diabetes.

File Number: 20870 

Disease: Autoimmune and Inflammation


IP Protection

Copyright: ©2010, The Regents of the University of California

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This innovation currently is not available for online licensing. Please contact Debbie Alexander at University of California System: University of California, San Francisco for more information.

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Debbie  Alexander Debbie Alexander

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