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 …

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 …

Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna

C Hinderer, P Bell, CH Vite, JP Louboutin… - … Therapy Methods & …, 2014 - cell.com
Adeno-associated virus serotype 9 (AAV9) vectors have recently been shown to transduce
cells throughout the central nervous system of nonhuman primates when injected into the …

In vivo selection yields AAV-B1 capsid for central nervous system and muscle gene therapy

SR Choudhury, Z Fitzpatrick, AF Harris, SA Maitland… - Molecular Therapy, 2016 - cell.com
Adeno-associated viral (AAV) vectors have shown promise as a platform for gene therapy of
neurological disorders. Achieving global gene delivery to the central nervous system (CNS) …

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) …

Widespread central nervous system gene transfer and silencing after systemic delivery of novel AAV-AS vector

SR Choudhury, AF Harris, DJ Cabral, AM Keeler… - Molecular Therapy, 2016 - cell.com
Effective gene delivery to the central nervous system (CNS) is vital for development of novel
gene therapies for neurological diseases. Adeno-associated virus (AAV) vectors have …

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 …

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 …

A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system

O Kondratov, L Kondratova, RJ Mandel, K Coleman… - Molecular Therapy, 2021 - cell.com
Non-human primates (NHPs) are a preferred animal model for optimizing adeno-associated
virus (AAV)-mediated CNS gene delivery protocols before clinical trials. In spite of its …

Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates

PA Lawlor, RJ Bland, A Mouravlev, D Young… - Molecular therapy, 2009 - cell.com
Adeno-associated viral (AAV) vectors have become the primary delivery agent for somatic
gene transfer into the central nervous system (CNS). To date, AAV-mediated gene delivery …