Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different …

C Burger, OS Gorbatyuk, MJ Velardo, CS Peden… - Molecular Therapy, 2004 - cell.com
Recombinant adeno-associated virus 2 (rAAV2) has been shown to deliver genes to
neurons effectively in the brain, retina, and spinal cord. The characterization of new AAV …

[HTML][HTML] AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges

L Kang, S Jin, J Wang, Z Lv, C Xin, C Tan… - Journal of Controlled …, 2023 - Elsevier
In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

CNS-restricted transduction and CRISPR/Cas9-mediated gene deletion with an engineered AAV vector

G Murlidharan, K Sakamoto, L Rao, T Corriher… - … Therapy-Nucleic Acids, 2016 - cell.com
Gene therapy using recombinant adeno-associated viral (AAV) vectors is emerging as a
promising approach to treat central nervous system disorders such as Spinal muscular …

[HTML][HTML] A combination of mutations enhances the neurotropism of AAV-2

J Xu, C Ma, C Bass, EF Terwilliger - Virology, 2005 - Elsevier
There is strong interest in developing practical strategies for gene delivery to the central
nervous system (CNS). Direct delivery into the brain or spinal cord is highly invasive as well …

Comparative transduction efficiency of AAV vector serotypes 1–6 in the substantia nigra and striatum of the primate brain

EA Markakis, KP Vives, J Bober, S Leichtle, C Leranth… - Molecular Therapy, 2010 - cell.com
Vectors derived from adeno-associated virus (AAV) are promising candidates for neural cell
transduction in vivo because they are nonpathogenic and achieve long-term transduction in …

Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates

HB Dodiya, T Bjorklund, J Stansell III, RJ Mandel… - Molecular Therapy, 2010 - cell.com
We examined the transduction efficiency of different adeno-associated virus (AAV) capsid
serotypes encoding for green fluorescent protein (GFP) flanked by AAV2 inverted terminal …

Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats

J Hordeaux, L Dubreil, J Deniaud, F Iacobelli… - Gene therapy, 2015 - nature.com
Intracerebral administration of recombinant adeno-associated vector (AAV) has been
performed in several clinical trials. However, delivery into the brain requires multiple …

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

KD Foust, E Nurre, CL Montgomery, A Hernandez… - Nature …, 2009 - nature.com
Delivery of genes to the brain and spinal cord across the blood-brain barrier (BBB) has not
yet been achieved. Here we show that adeno-associated virus (AAV) 9 injected …

Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model

R Song, K Pekrun, TA Khan, F Zhang, SP Paşca… - … Therapy-Methods & …, 2022 - cell.com
A limitation for recombinant adeno-associated virus (rAAV)-mediated gene transfer into the
central nervous system (CNS) is the low penetration of vectors across the human blood …

Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neurons

KD Foust, A Poirier, CA Pacak, RJ Mandel… - Human gene …, 2008 - liebertpub.com
Targeting lower motor neurons (LMNs) for gene delivery could be useful for disorders such
as spinal muscular atrophy and amyotrophic lateral sclerosis. LMNs reside in the ventral …