Overcoming the delivery problem for therapeutic genome editing: Current status and perspective of non-viral methods.

Overcoming the delivery problem for therapeutic genome editing: Current status and perspective of non-viral methods. Biomaterials. 2020 Aug 06;258:120282 Authors: Mashel TV, Tarakanchikova YV, Muslimov AR, Zyuzin MV, Timin AS, Lepik KV, Fehse B Abstract Besides its broad application in research and biotechnology, genome editing (GE) has great potential for clinical gene therapy, but delivery of GE tools remains a bottleneck. Whereas significant progress has been made in ex vivo GE delivery (e.g., by electroporation), establishment of efficient and safe in vivo delivery systems is still a challenge. Above and beyond standard vector requirements (safety, minimal/absent toxicity and immunogenicity, sufficient packaging capacity, targeting, straight and low-cost large-scale good manufacturing practice (GMP) production), GE delivery systems ideally use a hit-and-run principle to minimize off-targets as well as display of immunogenic peptides. Since currently used viral vectors do not fulfil all of these requirements, the broad variety of non-viral delivery platforms represents a promising alternative. This review provides a comprehensive analysis of the most relevant aspects of non-viral physical and chemical delivery methods in non-clinical studies and clinical trials, ranging from classic electroporation to advanced drug carriers that can transport GE tools in form of plasmid DNAs (pDNAs), mRNAs, and ribonucleoproteins (RNPs). For compa...
Source: Biomaterials - Category: Materials Science Authors: Tags: Biomaterials Source Type: research