Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain

CN Cearley, LH Vandenberghe, MK Parente… - Molecular therapy, 2008 - cell.com
A wide diversity of adeno-associated virus (AAV) structural proteins uncovered from latent
genomes in primate tissue has expanded the number of AAV vector serotypes, which can …

Systemic AAV vectors for widespread and targeted gene delivery in rodents

RC Challis, S Ravindra Kumar, KY Chan, C Challis… - Nature protocols, 2019 - nature.com
We recently developed adeno-associated virus (AAV) capsids to facilitate efficient and
noninvasive gene transfer to the central and peripheral nervous systems. However, a …

A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency

J Tordo, C O'Leary, ASLM Antunes, N Palomar… - Brain, 2018 - academic.oup.com
Recombinant adeno-associated viruses (AAVs) are popular in vivo gene transfer vehicles.
However, vector doses needed to achieve therapeutic effect are high and some target …

[HTML][HTML] Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse

DJ Schuster, JA Dykstra, MS Riedl, KF Kitto… - Frontiers in …, 2014 - frontiersin.org
Adeno-associated virus serotype 9 (AAV9)-mediated gene transfer has been reported in
central nervous system (CNS) and peripheral tissues. The current study compared the …

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

SJ Gray, V Matagne, L Bachaboina, S Yadav… - Molecular Therapy, 2011 - cell.com
Other labs have previously reported the ability of adeno-associated virus serotype 9 (AAV9)
to cross the blood-brain barrier (BBB). In this report, we carefully characterized variables that …

Gene therapy for the CNS using AAVs: the impact of systemic delivery by AAV9

J Saraiva, RJ Nobre, LP de Almeida - Journal of Controlled Release, 2016 - Elsevier
Several attempts have been made to discover the ideal vector for gene therapy in central
nervous system (CNS). Adeno-associated viruses (AAVs) are currently the preferred vehicle …

Selective and rapid uptake of adeno-associated virus type 2 in brain

JS Bartlett, RJ Samulski, TJ Mccown - Human gene therapy, 1998 - liebertpub.com
Recombinant adeno-associated virus (AAV) vectors effectively transfer and express foreign
genes in the brain. The transferred genes, however, are selectively expressed in neurons …

AAV-PHP. B administration results in a differential pattern of CNS biodistribution in non-human primates compared with mice

WA Liguore, JS Domire, D Button, Y Wang, BD Dufour… - Molecular Therapy, 2019 - cell.com
The ability of recombinant adeno-associated virus (AAV) to deliver transgenes to the CNS
has allowed for several advancements in the field of gene therapy to treat brain disorders …

Adeno-associated virus (AAV) vectors in the CNS

TJ McCown - Current gene therapy, 2005 - ingentaconnect.com
Adeno-associated virus (AAV) vectors exhibit a number of properties that have made this
vector system an excellent choice for both CNS gene therapy and basic neurobiological …

Rationally engineered AAV capsids improve transduction and volumetric spread in the CNS

NM Kanaan, RC Sellnow, SL Boye, B Coberly… - … Therapy-Nucleic Acids, 2017 - cell.com
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 …