Development of New Strategies Using Extracellular Vesicles Loaded with Exogenous Nucleic Acid.
Nicola Salvatore OreficePublished in: Pharmaceutics (2020)
Gene therapy is a therapeutic strategy of delivering foreign genetic material (encoding for an important protein) into a patient's target cell to replace a defective gene. Nucleic acids are embedded within the adeno-associated virus (AAVs) vectors; however, preexisting immunity to AAVs remains a significant concern that impairs their clinical application. Extracellular vesicles (EVs) hold great potential for therapeutic applications as vectors of nucleic acids due to their endogenous intercellular communication functions through their cargo delivery, including lipids and proteins. So far, small RNAs (siRNA and micro (mi)RNA) have been mainly loaded into EVs to treat several diseases, but the potential use of EVs to load and deliver exogenous plasmid DNA has not been thoroughly described. This review provides a comprehensive overview of the principal methodologies currently employed to load foreign genetic material into EVs, highlighting the need to find the most effective strategies for their successful clinical translations.
Keyphrases
- gene therapy
- nucleic acid
- genome wide
- cancer therapy
- drug delivery
- copy number
- escherichia coli
- single cell
- human health
- case report
- wound healing
- crispr cas
- dna methylation
- cell therapy
- single molecule
- cell free
- fatty acid
- risk assessment
- stem cells
- binding protein
- transcription factor
- circulating tumor
- climate change
- hyaluronic acid
- genome wide identification