Self-inactivating Transposase Plasmids for Genetic Engineering and Gene Therapy
Platform transposase/transposon plasmid for genetic engineering applications
Transposon-based gene delivery strategies offer multiple advantages over viral vector-based approaches, including reduced immunogenicity; reduced risk of insertional mutagenesis; the ability to deliver large transgenes and multiple genetic elements; and lower cost. The transposon and transposase are typically separately delivered to eliminate potential integrations of the transposase genes into the host genome. However, the two-component system leads to decreased efficiency and drives up cost and complexity. Dr. Michael Jensen and colleagues have designed a single plasmid that carries a self-inactivating transposase gene and a corresponding transposon. This system utilizes intrinsic cellular machinery to mediate self-inactivation of the transposase enzyme and improve safety. The plasmids have been validated in cell lines and primary human cells. This single component transposase system is a novel platform for non-viral gene therapy and genetic engineering.
- Non-viral gene therapy
- Genetic engineering
- Single component transposon-based, non-viral genetic engineering
- Reduced manufacturing costs
The global gene therapy market is expected to grow at a CAGR of 33.9% from 2018 to 2026. Though the market is dominated by viral vector-based delivery methods, unwanted immune responses associated with viral vectors constitute a major hurdle to market growth. Non-viral vector-based gene delivery has shown positive results in preclinical and clinical trials for various diseases. This segment is expected to be lucrative and projected to grow during the analysis period.
Preclinical in vitro and ex vivo
Michael Jensen, MD
Joshua Gustafson, PhD
Jeremy Bjelajac, PhD
Joseph Cheng, PhD
Kamila Gwiazda, PhD
To learn more about this technology, please email Kamya Rajaram.