Molecular brain, 2014-03, Vol.7 (1), p.17-17, Article 17
2014
Details
- Autor(en) / Beteiligte
- Titel
- Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons
- Ist Teil von
- Molecular brain, 2014-03, Vol.7 (1), p.17-17, Article 17
- Ort / Verlag
- England: BioMed Central Ltd
- Erscheinungsjahr
- 2014
- Link zum Volltext
- Quelle
- Free E-Journal (出版社公開部分のみ）
- Beschreibungen/Notizen
- Adeno-associated virus (AAV) vectors can deliver transgenes to diverse cell types and are therefore useful for basic research and gene therapy. Although AAV has many advantages over other viral vectors, its relatively small packaging capacity limits its use for delivering large genes. The available transgene size is further limited by the existence of additional elements in the expression cassette without which the gene expression level becomes much lower. By using alternative combinations of shorter elements, we generated a series of AAV expression cassettes and systematically evaluated their expression efficiency in neurons to maximize the transgene size available within the AAV packaging capacity while not compromising the transgene expression. We found that the newly developed smaller expression cassette shows comparable expression efficiency with an efficient vector generally used for strong gene expression. This new expression cassette will allow us to package larger transgenes without compromising expression efficiency.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 1756-6606eISSN: 1756-6606DOI: 10.1186/1756-6606-7-17Titel-ID: cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3975461
- Format
- –
- Schlagworte
- Adeno-associated virus, Animals, Brain - metabolism, Colleges & universities, Dependovirus - genetics, Dependovirus - physiology, Efficiency, Gene Expression, Genes, Genetic aspects, Genetic Techniques, Genetic Vectors - metabolism, Genomes, Growth hormones, Health aspects, HEK293 Cells, Hepatitis, Humans, Male, Methodology, Mice, Mice, Inbred C57BL, Mutagenesis, Neurons, Neurons - metabolism, Optimization, Polyadenylation - genetics, Proteins, Rats, Regulatory Sequences, Nucleic Acid - genetics, Rodents, Science, Sequence Deletion, Transgenes - genetics, Virus Assembly - physiology