PMID- 23517473
OWN - NLM
STAT- MEDLINE
DCOM- 20131231
LR  - 20211021
IS  - 1557-7422 (Electronic)
IS  - 1043-0342 (Print)
IS  - 1043-0342 (Linking)
VI  - 24
IP  - 5
DP  - 2013 May
TI  - Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into 
      the cerebrospinal fluid of nonhuman primates.
PG  - 526-32
LID - 10.1089/hum.2013.005 [doi]
AB  - 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 transduction throughout cortex and spinal cord. 
      Transduction efficiency was severely limited, however, by the presence of 
      circulating anti-AAV antibodies. Accordingly, we compared AAV9 to a related 
      serotype, AAV7, which has a high capsid homology. CM infusion of either AAV7 or 
      AAV9 directed high level of cell transduction with similar patterns of 
      distribution throughout brain cortex and along the spinal cord. Dorsal root 
      ganglia and corticospinal tracts were also transduced. Both astrocytes and 
      neurons were transduced. Interestingly, little transduction was observed in 
      peripheral organs. Our results indicate that intrathecal delivery of either AAV7 
      or AAV9 directs a robust and widespread cellular transduction in the central 
      nervous system and other peripheral neural structures.
FAU - Samaranch, Lluis
AU  - Samaranch L
AD  - Department of Neurological Surgery, University of California San Francisco, CA 
      94103, USA.
FAU - Salegio, Ernesto A
AU  - Salegio EA
FAU - San Sebastian, Waldy
AU  - San Sebastian W
FAU - Kells, Adrian P
AU  - Kells AP
FAU - Bringas, John R
AU  - Bringas JR
FAU - Forsayeth, John
AU  - Forsayeth J
FAU - Bankiewicz, Krystof S
AU  - Bankiewicz KS
LA  - eng
GR  - R01NS073940-01/NS/NINDS NIH HHS/United States
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
DEP - 20130502
PL  - United States
TA  - Hum Gene Ther
JT  - Human gene therapy
JID - 9008950
RN  - 147336-22-9 (Green Fluorescent Proteins)
SB  - IM
MH  - Animals
MH  - Astrocytes/pathology/virology
MH  - Cerebral Cortex/pathology/virology
MH  - Dependovirus/*genetics
MH  - Ganglia, Spinal/metabolism/pathology/virology
MH  - Gene Transfer Techniques/*adverse effects
MH  - Genetic Vectors/*adverse effects/cerebrospinal fluid
MH  - Green Fluorescent Proteins/genetics
MH  - Macaca/*genetics/virology
MH  - Motor Neurons/metabolism/pathology/virology
MH  - Spinal Cord/pathology/virology
MH  - *Transduction, Genetic
PMC - PMC3655626
EDAT- 2013/03/23 06:00
MHDA- 2014/01/01 06:00
PMCR- 2014/05/01
CRDT- 2013/03/23 06:00
PHST- 2013/03/23 06:00 [entrez]
PHST- 2013/03/23 06:00 [pubmed]
PHST- 2014/01/01 06:00 [medline]
PHST- 2014/05/01 00:00 [pmc-release]
AID - 10.1089/hum.2013.005 [pii]
AID - 10.1089/hum.2013.005 [doi]
PST - ppublish
SO  - Hum Gene Ther. 2013 May;24(5):526-32. doi: 10.1089/hum.2013.005. Epub 2013 May 2.