Ontology highlight
ABSTRACT:
SUBMITTER: Kanaan NM
PROVIDER: S-EPMC5503098 | biostudies-literature | 2017 Sep
REPOSITORIES: biostudies-literature
Kanaan Nicholas M NM Sellnow Rhyomi C RC Boye Sanford L SL Coberly Ben B Bennett Antonette A Agbandje-McKenna Mavis M Sortwell Caryl E CE Hauswirth William W WW Boye Shannon E SE Manfredsson Fredric P FP
Molecular therapy. Nucleic acids 20170621
Adeno-associated virus (AAV) is the most common vector for clinical gene therapy of the CNS. This popularity originates from a high safety record and the longevity of transgene expression in neurons. Nevertheless, clinical efficacy for CNS indications is lacking, and one reason for this is the relatively limited spread and transduction efficacy in large regions of the human brain. Using rationally designed modifications of the capsid, novel AAV capsids have been generated that improve intracellu ...[more]