Tech ID: 32860 / UC Case 2022-143-0

Patent Status

Country Type Number Dated Case
Patent Cooperation Treaty Published Application WO 2023/250324 12/28/2023 2022-143

Additional Patent Pending

Brief Description

Human diseases that follow a dominant negative inheritance pattern present a great challenge for treatment using gene therapy methods. In such cases, a copy of an allele is inherited from each parent: one is a pathogenic allele causing a disease phenotype (e.g., by exerting a toxic, gain-of-function effect) and the other is a wild-type (non-pathogenic) allele. Allele-specific targeting is especially important when the wild-type allele is crucial to normal function, e.g., the wild-type allele encodes a protein whose function is critical. There is therefore a need for compositions and methods of allele-specific gene editing.


UC Berkeley researchers have created methods and systems for reducing the level of an RNA transcript from a target nucleic acid in an allele-specific manner. Such systems and methods can be used to treat a disease that results from or is caused by a toxic gain-of-function protein. 


Suggested uses

  • Reducing level of RNA transcript from a target nucleic acid
  • Treatment of diseases resulting from or caused by a toxic gain-of-function protein

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  • Lareau, Liana F.

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