Gene transfer by adeno-associated virus vectors into the central nervous system

X Xiao, J Li, TJ McCown, RJ Samulski - Experimental neurology, 1997 - Elsevier
Adeno-associated virus (AAV) vectors are derived from a nonpathogenic and defective human
parvovirus. Although currently unable to display the integration specificity featured by its …

Adeno-associated virus (AAV) gene therapy for neurological disease

MS Weinberg, RJ Samulski, TJ McCown - Neuropharmacology, 2013 - Elsevier
Diseases of the central nervous system (CNS) have provided enormous opportunities for
the therapeutic application of viral vector gene transfer. Adeno-associated virus (AAV) has …

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 …

Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector

TJ McCown, X Xiao, J Li, GR Breese, RJ Samulski - Brain research, 1996 - Elsevier
Safe, long-term gene expression is a primary criteria for effective gene therapy in the brain,
so studies were initiated to evaluate adeno-associated virus (AAV) vector transfer of a …

Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary …

…, J Coleman, MD Ehlers, MJ Zylka, TJ McCown… - Human gene …, 2011 - liebertpub.com
… made by McCown and colleagues and Klein and colleagues that the CMV promoter is
silenced over time in the hippocampus after direct intracranial injection (McCown et al., 1996; …

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, …

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

SJ Gray, S Nagabhushan Kalburgi, TJ McCown… - Gene therapy, 2013 - nature.com
Injection of adeno-associated virus (AAV) into the cerebrospinal fluid (CSF) offers a means
to achieve widespread transgene delivery to the central nervous system, where the doses …

[HTML][HTML] Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles

…, S Leichtle, DE Redmond Jr, TJ McCown… - Molecular Therapy, 2008 - cell.com
We report a DNA shuffling–based approach for developing cell type–specific vectors through
directed evolution. Capsid genomes of adeno-associated virus (AAV) serotypes 1–9 were …

[HTML][HTML] Behavioral differences between neonatal and adult 6-hydroxydopamine-treated rats to dopamine agonists: relevance to neurological symptoms in clinical …

GR Breese, AA Baumeister, TJ McCOWN… - The Journal of …, 1984 - ncbi.nlm.nih.gov
Administration of L-dopa or apomorphine to neonatal and adult 6-hydroxydopamine (6-OHDA)-treated
rats resulted in different behavioral responses depending on the age at which …

[HTML][HTML] Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood–brain barrier (BBB)

…, HE Criswell, SC Nicolson, RJ Samulski, TJ McCown - Molecular Therapy, 2010 - cell.com
… First, WT adenovirus would need to be injected directly into the CNS, but soon after direct
injection adenovirus toxicity would kill many if not most of the infected cells (TJ McCown and …