PMID- 26283910
OWN - NLM
STAT- PubMed-not-MEDLINE
DCOM- 20150818
LR  - 20201001
IS  - 1662-5099 (Print)
IS  - 1662-5099 (Electronic)
IS  - 1662-5099 (Linking)
VI  - 8
DP  - 2015
TI  - Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and 
      the spinal cord of neonatal mice.
PG  - 36
LID - 10.3389/fnmol.2015.00036 [doi]
LID - 36
AB  - Systemic delivery of self-complementary (sc) adeno-associated-virus vector of 
      serotype 9 (AAV9) was recently shown to provide robust and widespread gene 
      transfer to the central nervous system (CNS), opening new avenues for practical, 
      and non-invasive gene therapy of neurological diseases. More recently, AAV of 
      serotype rh10 (AAVrh10) was also found highly efficient to mediate CNS 
      transduction after intravenous administration in mice. However, only a few 
      studies compared AAV9 and AAVrh10 efficiencies, particularly in the spinal cord. 
      In this study, we compared the transduction capabilities of AAV9 and AAVrh10 in 
      the brain, the spinal cord, and the peripheral nervous system (PNS) after 
      intravenous delivery in neonatal mice. As reported in previous studies, AAVrh10 
      achieved either similar or higher transduction than AAV9 in all the examined 
      brain regions. The superiority of AAVrh10 over AAV9 appeared statistically 
      significant only in the medulla and the cerebellum, but a clear trend was also 
      observed in other structures like the hippocampus or the cortex. In contrast to 
      previous studies, we found that AAVrh10 was more efficient than AAV9 for 
      transduction of the dorsal spinal cord and the lower motor neurons (MNs). 
      However, differences between the two serotypes appeared mainly significant at low 
      dose, and surprisingly, increasing the dose did not improve AAVrh10 distribution 
      in the spinal cord, in contrary to AAV9. Similar dose-related differences between 
      transduction efficiency of the two serotypes were also observed in the sciatic 
      nerve. These findings suggest differences in the transduction mechanisms of these 
      two serotypes, which both hold great promise for gene therapy of neurological 
      diseases.
FAU - Tanguy, Yannick
AU  - Tanguy Y
AD  - Center of Research on Myology, FRE 3617 Centre National de la Recherche 
      Scientifique, UMRS 974 INSERM, French Institute of Myology, Pierre and Marie 
      Curie University Paris, France.
FAU - Biferi, Maria G
AU  - Biferi MG
AD  - Center of Research on Myology, FRE 3617 Centre National de la Recherche 
      Scientifique, UMRS 974 INSERM, French Institute of Myology, Pierre and Marie 
      Curie University Paris, France.
FAU - Besse, Aurore
AU  - Besse A
AD  - Center of Research on Myology, FRE 3617 Centre National de la Recherche 
      Scientifique, UMRS 974 INSERM, French Institute of Myology, Pierre and Marie 
      Curie University Paris, France.
FAU - Astord, Stephanie
AU  - Astord S
AD  - Center of Research on Myology, FRE 3617 Centre National de la Recherche 
      Scientifique, UMRS 974 INSERM, French Institute of Myology, Pierre and Marie 
      Curie University Paris, France.
FAU - Cohen-Tannoudji, Mathilde
AU  - Cohen-Tannoudji M
AD  - Center of Research on Myology, FRE 3617 Centre National de la Recherche 
      Scientifique, UMRS 974 INSERM, French Institute of Myology, Pierre and Marie 
      Curie University Paris, France.
FAU - Marais, Thibaut
AU  - Marais T
AD  - Center of Research on Myology, FRE 3617 Centre National de la Recherche 
      Scientifique, UMRS 974 INSERM, French Institute of Myology, Pierre and Marie 
      Curie University Paris, France.
FAU - Barkats, Martine
AU  - Barkats M
AD  - Center of Research on Myology, FRE 3617 Centre National de la Recherche 
      Scientifique, UMRS 974 INSERM, French Institute of Myology, Pierre and Marie 
      Curie University Paris, France.
LA  - eng
PT  - Journal Article
DEP - 20150728
PL  - Switzerland
TA  - Front Mol Neurosci
JT  - Frontiers in molecular neuroscience
JID - 101477914
PMC - PMC4516891
OTO - NOTNLM
OT  - AAV9
OT  - AAVrh10
OT  - SMA
OT  - adeno-associated virus
OT  - central nervous system
OT  - gene therapy
OT  - motor neuron
OT  - peripheral nervous system
EDAT- 2015/08/19 06:00
MHDA- 2015/08/19 06:01
PMCR- 2015/01/01
CRDT- 2015/08/19 06:00
PHST- 2015/05/14 00:00 [received]
PHST- 2015/07/06 00:00 [accepted]
PHST- 2015/08/19 06:00 [entrez]
PHST- 2015/08/19 06:00 [pubmed]
PHST- 2015/08/19 06:01 [medline]
PHST- 2015/01/01 00:00 [pmc-release]
AID - 10.3389/fnmol.2015.00036 [doi]
PST - epublish
SO  - Front Mol Neurosci. 2015 Jul 28;8:36. doi: 10.3389/fnmol.2015.00036. eCollection 
      2015.