Journal
TRENDS IN BIOTECHNOLOGY
Volume 33, Issue 12, Pages 777-790Publisher
ELSEVIER SCIENCE LONDON
DOI: 10.1016/j.tibtech.2015.09.008
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Funding
- LOEWE Center for Cell and Gene Therapy Frankfurt - Hessisches Ministerium fur Wissenschaft und Kunst [III L4-518/17.004]
- Center for Molecular Medicine Cologne (CMMC)
- German Center for Infection Research (DZIF)
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Recent progress in gene transfer technology enables the delivery of genes precisely to the application-relevant cell type ex vivo on cultivated primary cells or in vivo on local or systemic administration. Gene vectors based on lentiviruses or adeno-associated viruses can be engineered such that they use a cell surface marker of choice for cell entry instead of their natural receptors. Binding to the surface marker is mediated by a targeting ligand displayed on the vector particle surface, which can be a peptide, single-chain antibody, or designed ankyrin repeat protein. Examples include vectors that deliver genes to specialized endothelial cells or lymphocytes, tumor cells, or particular cells of the nervous system with potential applications in gene function studies and molecular medicine.
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