RETROTRANSPOSON-BASED DELIVERY VEHICLE AND METHODS OF USE THEREOF

Tech ID: 29232 / UC Case 2018-120-0

Patent Status

Country Type Number Dated Case
Japan Published Application 2021-530212 11/11/2021 2018-120
United States Of America Published Application 20210285009 09/16/2021 2018-120
European Patent Office Published Application 3821012 05/19/2021 2018-120
China Published Application CN112513270A 03/16/2021 2018-120
Canada Published Application WO 2020/014528 01/16/2020 2018-120
Patent Cooperation Treaty Published Application WO2020014528 01/16/2020 2018-120
 

Brief Description


Gene therapy delivery generally falls into two main categories: viral-mediated and non-viral mediated delivery. Viral-mediated integrative approaches are most commonly used in dividing cells, where delivery is mediated, e.g., through the use of lentiviruses and retroviruses engineered to carry therapeutic DNA into cells. Such viruses have a number of drawbacks, e.g., disruption of the cell's function, the cell’s own machinery may silence expression and payload size limitations. Whereas class II transposons such as piggyBac, Sleeping Beauty, and Tol2 can integrate larger payloads; however, such transposons have a tendency to integrate in areas where active transcription is occurring. Therefore, there is a need in the art for delivery vehicles that provide for delivery of larger coding regions. 

 

UC Berkeley researchers have developed a gene delivery system that has a nucleotide sequence encoding an R2 retrotransposon R2 polypeptide that is able to deliver one or more gene products of interest to a eukaryotic cell.


Suggested uses


  • gene therapy
  • gene delivery

Advantages


  • Very small delivery system capable of delivery larger payloads of larger cDNA, regulatory elements, or multiple genes 

 

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Other Information

Keywords

gene, delivery

Categorized As