Open AccessAdeno-associated Virus (AAV) Capsid Chimeras with Enhanced Infectivity Reveal a Core Element in the AAV Genome Critical for both Cell Transduction and Capsid Assembly
Author(s) -
Lydia Viney,
Tilmann Bürckstümmer,
Courtnee Eddington,
Mario Mietzsch,
Modassir Choudhry,
Tom Henley,
Mavis AgbandjeMcKenna
Publication year - 2021
Publication title -
journal of virology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.617
H-Index - 292
eISSN - 1070-6321
pISSN - 0022-538X
DOI - 10.1128/jvi.02023-20
Subject(s) - capsid , adeno associated virus , biology , transduction (biophysics) , genetic enhancement , virology , gene delivery , virus , transgene , infectivity , gene , genetics , vector (molecular biology) , recombinant dna , biochemistry
A major hurdle to the therapeutic potential of AAV in gene therapy lies in achieving clinically meaningful AAV doses, and secondarily, the ability to manufacture commercially viable titers of AAV to support this. By virtue of neutralizing antibodies against AAV that impede patient repeat dosing, the dose of AAV for in vivo gene delivery has been high, which has resulted in unfortunate recent safety concerns and deaths in patients given higher-dose AAV gene therapy.