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Novel Gene Therapeutic for Degrading Misfolded-Proteins


Brief Description

Penn researchers have identified a specific system using SUMOylation and ubiquitination to dramatically reduce protein aggregates and target the misfolded proteins for degradation, thus clearing the underlying cause of certain neurodegenerative diseases. 


Docket #  14-7104 



Technology Overview

  The accumulation of misfolded proteins compromises cellular function, resulting in debilitating diseases. There is no current effective therapeutic that prevents this underlying disease causation.

  Penn researcher, Dr. Xiaolu Yang, has identified a novel therapeutic peptide, deliverable by targeted gene therapy, which promotes the degradation of various misfolded proteins in the mammalian cell. Targeting of this pathway for treatment or protection from neurodegenerative diseases is an unexplored therapeutic area. Dr. Yang has identified additional analogous targets and is currently testing in vivo efficacy in neurodegenerative models.



Dr. Xiaolu Yang

The Yang Lab studies the molecular and cellular mechanisms that protect against major diseases, including cancer and neurodegeneration. 

State of Development

In vivo proof of concept


Intellectual Property

provisional patent


Reference Media

Mol Cell. 2014 Jul 3;55(1):15-30. doi: 10.1016/j.molcel.2014.04.030.



Desired partnerships

• Research collaboration

• Sponsored research


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Keywords: Neurodegenerative Disease, Life Sciences

Patent Information:
For Information, Contact:
Linara Axanova
Associate Director, PSOM Licensing Group
University of Pennsylvania
Xiaolu Yang
Neurodegenerative Diseases