PMID- 31146917
OWN - NLM
STAT- MEDLINE
DCOM- 20200629
LR  - 20200629
IS  - 1090-2104 (Electronic)
IS  - 0006-291X (Linking)
VI  - 515
IP  - 1
DP  - 2019 Jul 12
TI  - Gene delivery to cone photoreceptors by subretinal injection of rAAV2/6 in the 
      mouse retina.
PG  - 222-227
LID - S0006-291X(19)31003-4 [pii]
LID - 10.1016/j.bbrc.2019.05.117 [doi]
AB  - Adeno-associated virus (AAV) has been studied as a safe delivery tool for gene 
      therapy of retinal blinding diseases such as Leber's congenital amaurosis (LCA). 
      The tropism of recombinant AAV (rAAV) including its specificity and efficiency in 
      targeting retinal cell types has been studied with native or engineered capsids, 
      along with specific promoters. However, one of the rAAV serotypes, rAAV2/6, has 
      not been well-studied based on a report of low infection efficiency in the 
      retina. We investigated the tropism of several rAAVs by subretinal injection in 
      the adult mouse and found that rAAV2/6 predominantly infected cone photoreceptors 
      including the main spectral type. Our data suggest that subretinal injection with 
      rAAV2/6 may provide both an efficacious and specific means of gene delivery to 
      cone photoreceptors in murine retinas.
CI  - Copyright (c) 2019. Published by Elsevier Inc.
FAU - Hori, Tesshu
AU  - Hori T
AD  - Graduate School of Pharmacy, Ritsumeikan University, Kusatsu, Shiga, Japan; 
      College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan.
FAU - Fukutome, Masashi
AU  - Fukutome M
AD  - College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan; Graduate School of Life Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan.
FAU - Maejima, Chiseto
AU  - Maejima C
AD  - College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan.
FAU - Matsushima, Hiroki
AU  - Matsushima H
AD  - Graduate School of Pharmacy, Ritsumeikan University, Kusatsu, Shiga, Japan; 
      College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan.
FAU - Kobayashi, Kensuke
AU  - Kobayashi K
AD  - College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan; Graduate School of Life Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan.
FAU - Kitazawa, Soichiro
AU  - Kitazawa S
AD  - College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan.
FAU - Kitahara, Ryo
AU  - Kitahara R
AD  - College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan; Graduate School of Life Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan.
FAU - Kitano, Katsunori
AU  - Kitano K
AD  - College of Information Science and Engineering, Ritsumeikan University, Kusatsu, 
      Shiga, Japan; Center for Systems Vision Science, Research Organization of Science 
      and Technology, Ritsumeikan University, Kusatsu, Shiga, Japan.
FAU - Kobayashi, Kenta
AU  - Kobayashi K
AD  - Section of Viral Vector Development, National Institute for Physiological 
      Sciences, Okazaki, Aichi, Japan.
FAU - Moritoh, Satoru
AU  - Moritoh S
AD  - College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan; Center for Systems Vision Science, Research Organization of Science and 
      Technology, Ritsumeikan University, Kusatsu, Shiga, Japan.
FAU - Koike, Chieko
AU  - Koike C
AD  - College of Pharmaceutical Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan; Graduate School of Life Sciences, Ritsumeikan University, Kusatsu, Shiga, 
      Japan; Center for Systems Vision Science, Research Organization of Science and 
      Technology, Ritsumeikan University, Kusatsu, Shiga, Japan. Electronic address: 
      koike@fc.ritsumei.ac.jp.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20190527
PL  - United States
TA  - Biochem Biophys Res Commun
JT  - Biochemical and biophysical research communications
JID - 0372516
RN  - 0 (Opsins)
SB  - IM
MH  - Animals
MH  - Dependovirus/*genetics
MH  - Genetic Therapy/*methods
MH  - Genetic Vectors/administration & dosage/*genetics
MH  - Injections
MH  - Leber Congenital Amaurosis/genetics/therapy
MH  - Mice, 129 Strain
MH  - Opsins/genetics/metabolism
MH  - Retina/*metabolism/virology
MH  - Retinal Cone Photoreceptor Cells/*metabolism/virology
MH  - Retinal Diseases/genetics/*therapy
MH  - Treatment Outcome
OTO - NOTNLM
OT  - Adeno-associated virus
OT  - Cone photoreceptors
OT  - Gene therapy
OT  - The mammalian retina
EDAT- 2019/05/31 06:00
MHDA- 2020/07/01 06:00
CRDT- 2019/06/01 06:00
PHST- 2019/05/05 00:00 [received]
PHST- 2019/05/17 00:00 [accepted]
PHST- 2019/05/31 06:00 [pubmed]
PHST- 2020/07/01 06:00 [medline]
PHST- 2019/06/01 06:00 [entrez]
AID - S0006-291X(19)31003-4 [pii]
AID - 10.1016/j.bbrc.2019.05.117 [doi]
PST - ppublish
SO  - Biochem Biophys Res Commun. 2019 Jul 12;515(1):222-227. doi: 
      10.1016/j.bbrc.2019.05.117. Epub 2019 May 27.