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 …

Efficient retrograde transport of adeno-associated virus type 8 to spinal cord and dorsal root ganglion after vector delivery in muscle

H Zheng, C Qiao, CH Wang, J Li, J Li, Z Yuan… - Human gene …, 2010 - liebertpub.com
The peripheral nervous system (PNS), including peripheral nerves and dorsal root ganglion
(DRG), is involved in numerous neurological disorders, such as peripheral neuropathies …

Widespread and efficient transduction of spinal cord and brain following neonatal AAV injection and potential disease modifying effect in ALS mice

JI Ayers, S Fromholt, O Sinyavskaya, Z Siemienski… - Molecular Therapy, 2015 - cell.com
The architecture of the spinal cord makes efficient delivery of recombinant adeno-associated
virus (rAAV) vectors throughout the neuraxis challenging. We describe a paradigm in which …

Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector

M Kudo, S Wupuer, M Fujiwara, Y Saito… - … therapy Methods & …, 2021 - cell.com
Adeno-associated virus 6 (AAV6) has been proposed as a potential vector candidate for
specific gene expression in pain-related dorsal root ganglion (DRG) neurons, but this has …

Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an adeno-associated virus vector expressing human SMN

C Hinderer, N Katz, EL Buza, C Dyer, T Goode… - Human gene …, 2018 - liebertpub.com
Neurotropic adeno-associated virus (AAV) serotypes such as AAV9 have been
demonstrated to transduce spinal alpha motor neurons when administered intravenously (iv) …

Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery

BR Snyder, SJ Gray, ET Quach, JW Huang… - Human gene …, 2011 - liebertpub.com
Gene therapy for motor neuron diseases requires efficient gene delivery to motor neurons
(MNs) throughout the spinal cord and brainstem. The present study compared adeno …

Intracerebroventricular injection of adeno-associated virus 6 and 9 vectors for cell type–specific transgene expression in the spinal cord

E Dirren, CL Towne, V Setola, DE Redmond Jr… - Human gene …, 2014 - liebertpub.com
In the context of motoneuron diseases, gene delivery as an experimental or therapeutic
approach is hindered by the challenge to specifically target cell populations that are widely …

Translational feasibility of lumbar puncture for intrathecal AAV administration

C Hinderer, N Katz, C Dyer, T Goode… - … Therapy Methods & …, 2020 - cell.com
Preclinical studies have demonstrated that a single injection of an adeno-associated virus
(AAV) vector into the cerebrospinal fluid (CSF) can achieve widespread gene transfer …

Differential adeno-associated virus mediated gene transfer to sensory neurons following intrathecal delivery by direct lumbar puncture

L Vulchanova, DJ Schuster, LR Belur… - Molecular …, 2010 - journals.sagepub.com
Background: Neuronal transduction by adeno-associated viral (AAV) vectors has been
demonstrated in cortex, brainstem, cerebellum, and sensory ganglia. Intrathecal delivery of …

Recombinant adeno-associated virus serotype 6 (rAAV2/6)-mediated gene transfer to nociceptive neurons through different routes of delivery

C Towne, M Pertin, AT Beggah, P Aebischer… - Molecular …, 2009 - journals.sagepub.com
Background Gene transfer to nociceptive neurons of the dorsal root ganglia (DRG) is a
promising approach to dissect mechanisms of pain in rodents and is a potential therapeutic …