Intra-CSF AAV9 and AAVrh10 administration in nonhuman primates: promising routes and vectors for which neurological diseases?

K Bey, J Deniaud, L Dubreil, B Joussemet… - … Therapy Methods & …, 2020 - cell.com
The identification of the most efficient method for whole central nervous system targeting that
is translatable to humans and the safest route of adeno-associated virus (AAV) …

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 …

MR-guided parenchymal delivery of adeno-associated viral vector serotype 5 in non-human primate brain

L Samaranch, B Blits, W San Sebastian, P Hadaczek… - Gene therapy, 2017 - nature.com
The present study was designed to characterize transduction of non-human primate brain
and spinal cord with AAV5 viral vector after parenchymal delivery. AAV5-CAG-GFP (1× 10 …

Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery

KL Pietersz, RM Martier, MS Baatje, JM Liefhebber… - Gene therapy, 2021 - nature.com
Various administration routes of adeno-associated virus (AAV)-based gene therapy have
been examined to target the central nervous system to answer the question what the most …

Potent spinal parenchymal AAV9-mediated gene delivery by subpial injection in adult rats and pigs

A Miyanohara, K Kamizato, S Juhas, J Juhasova… - … Therapy Methods & …, 2016 - cell.com
Effective in vivo use of adeno-associated virus (AAV)-based vectors to achieve gene-specific
silencing or upregulation in the central nervous system has been limited by the inability to …

Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies

RM Bailey, A Rozenberg, SJ Gray - Brain research, 2020 - Elsevier
Gene therapy clinical trials for neurological disorders are ongoing using intrathecal injection
of adeno-associated virus (AAV) vector directly into the cerebral spinal fluid. Preliminary …

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 …

The advent of AAV9 expands applications for brain and spinal cord gene delivery

RD Dayton, DB Wang, RL Klein - Expert opinion on biological …, 2012 - Taylor & Francis
Introduction: Straightforward studies compared adeno-associated virus (AAV) serotypes to
determine the most appropriate one for robust expression in the CNS. AAV9 was efficient …

AAV9: a potential blood-brain barrier buster

FP Manfredsson, AC Rising, RJ Mandel - Molecular Therapy, 2009 - cell.com
© The American Society of Gene Therapy commentary species, probably as a result of
differential expression of receptors across species. For instance, direct administration of …

Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates

L Samaranch, EA Salegio, W San Sebastian… - Human gene …, 2013 - liebertpub.com
The present study builds on previous work showing that infusion of adeno-associated virus
type 9 (AAV9) into the cisterna magna (CM) of nonhuman primates resulted in widespread …