Ultrasound Mediated Non-viral Gene and Drug Delivery
A novel, minimally invasive, non-viral method of using ultrasound to deliver drugs, proteins or nucleic acid compounds to tissues of interest in large animal models
Non-viral gene therapy approaches have several advantages over viral vector-based delivery, including non-host genome destruction, better spatial and temporal control, greater flexibility in vector construction and design and lower immunopathogenicity. Ultrasound mediated gene delivery (UMGD) has long been recognized as a method to perform minimally invasive, non-viral gene transfer. The technique has been utilized to deliver genes and therapeutic agents to several tissues. However, this method has been associated with poor delivery efficiency in laboratory animals. Dr. Carol Miao and colleagues have developed methods and devices to improve delivery efficiency associated with UMGD and extended its application to large animals. Improved gene transfection efficiency was achieved using novel ultrasound transducer designs and beam patterns, and ultrasound protocols were modified to improve treatment safety. Combining this methodology with minimally invasive transhepatic venous approach, the researchers achieved transcutaneous UMGD of luciferase into the liver of pigs. They detected focal regions of >105 RLU/mg protein with minimal tissue damage, with over a 100-fold increase in luciferase activity relative to untreated lobes. Most significantly, therapeutic levels of gene expression are achieved following transcutaneous UMGD of factor VIII plasmids into the liver of large animal models. Non-viral, ultrasound mediated therapeutic gene delivery methods and devices constitute a promising new strategy to treat human patients with genetic diseases.
- In vivo, non-viral delivery of nucleic acids, proteins, oligos and drugs
- Targeted delivery into large animal models
- Method to enable in vivo CRISPR/Cas based gene editing
- Minimally invasive delivery method
- Targeted or tissue-specific delivery
- Nuclear delivery of cargo
- Lower immune activation compared to viral vector mediated delivery
The global gene therapy market was valued at USD $536.43 million in 2018 and is expected to reach USD $5.55 billion by 2026 growing at a CAGR of 33.9%. 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.
Therapeutic delivery method
Preclinical in vivo
Carol Miao, PhD
Feng Zhang, PhD
To learn more about this technology, please email Kamya Rajaram.