Project Description

Composed of neurons, nervous system constitutes the marvel and also the most important part of life, which transmits signals and coordinates actions. Neurological damage and neurodegenerative diseases are intractable threats to human life. However, extant therapies have varied defects which, to a large extent, hinder the treatments to these problems. For molecular medication, a highly selective semipermeable membrane called blood-brain barrier (BBB) separates the circulating blood from the brain and extracellular fluid in the central nervous system (CNS), posing a major obstacle for drugs to function in the brain. Even though some drugs are capable of crossing this barrier, its non-specificity becomes the major concern of undermining CNS or causing inestimable side effects. Concurrently, viral gene therapy, for its immature technology and also the risk of eliciting immune response, is negated to be a persuasive treatment strategy.

Therefore, this year, we decided to engineer human embryonic kidney cells (HEK293T) to produce desired exosomes (kind of cell derived vesicles with diameter between 30 nm ~100nm) that are naturally capable of passing BBB. Our project involves mainly 4 parts: RNA packaging, exosome production boosting, exosome delivery and target cell characterization. After engineering, the resultant HEK293T cells may produce exosomes in large quantity and the exosomes contain therapeutic RNA molecules which can be translated in target neurons to strengthen either neuroregeneration or cure or alleviate neurodegenerative diseases. Moreover, the exosomes produced are also conferred an increased ability to specifically target neurons.