PMID- 30345694
OWN - NLM
STAT- MEDLINE
DCOM- 20200605
LR  - 20200605
IS  - 1442-9071 (Electronic)
IS  - 1442-6404 (Linking)
VI  - 47
IP  - 4
DP  - 2019 May
TI  - Gene therapy and the adeno-associated virus in the treatment of genetic and 
      acquired ophthalmic diseases in humans: Trials, future directions and safety 
      considerations.
PG  - 521-536
LID - 10.1111/ceo.13416 [doi]
AB  - Voretigene neparvovec-rzyl was recently approved for the treatment of Leber 
      congenital amaurosis, and the use of gene therapy for eye disease is attracting 
      even greater interest. The eye has immune privileged status, is easily 
      accessible, requires a reduced dosage of therapy due to its size and is highly 
      compartmentalized, significantly reducing systemic spread. Adeno-associated virus 
      (AAV), with its low pathogenicity, prolonged expression profile and ability to 
      transduce multiple cell types, has become the leading gene therapy vector. Target 
      diseases have moved beyond currently untreatable inherited dystrophies to common, 
      partially treatable acquired conditions such as exudative age-related macular 
      degeneration and glaucoma, but use of the technology in these conditions imposes 
      added obligations for caution in vector design. This review discusses the current 
      status of AAV gene therapy trials in genetic and acquired ocular diseases, and 
      explores new scientific developments, which could help ensure effective and safe 
      use of the therapy in the future.
CI  - (c) 2018 Royal Australian and New Zealand College of Ophthalmologists.
FAU - Ramlogan-Steel, Charmaine A
AU  - Ramlogan-Steel CA
AD  - LVF Ophthalmology Research Centre, Translational Research Institute, Brisbane, 
      Australia.
AD  - Greenslopes Clinical School, Faculty of Medicine, University of Queensland, 
      Greenslopes Hospital, Brisbane, Australia.
AD  - Medical and Applied Science, Central Queensland University, School of Health, 
      Rockhampton, Australia.
FAU - Murali, Aparna
AU  - Murali A
AUID- ORCID: 0000-0002-1567-3430
AD  - LVF Ophthalmology Research Centre, Translational Research Institute, Brisbane, 
      Australia.
AD  - Greenslopes Clinical School, Faculty of Medicine, University of Queensland, 
      Greenslopes Hospital, Brisbane, Australia.
FAU - Andrzejewski, Slawomir
AU  - Andrzejewski S
AD  - LVF Ophthalmology Research Centre, Translational Research Institute, Brisbane, 
      Australia.
AD  - Greenslopes Clinical School, Faculty of Medicine, University of Queensland, 
      Greenslopes Hospital, Brisbane, Australia.
FAU - Dhungel, Bijay
AU  - Dhungel B
AD  - Greenslopes Clinical School, Faculty of Medicine, University of Queensland, 
      Greenslopes Hospital, Brisbane, Australia.
FAU - Steel, Jason C
AU  - Steel JC
AD  - Medical and Applied Science, Central Queensland University, School of Health, 
      Rockhampton, Australia.
FAU - Layton, Christopher J
AU  - Layton CJ
AUID- ORCID: 0000-0001-6933-9991
AD  - LVF Ophthalmology Research Centre, Translational Research Institute, Brisbane, 
      Australia.
AD  - Greenslopes Clinical School, Faculty of Medicine, University of Queensland, 
      Greenslopes Hospital, Brisbane, Australia.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20190102
PL  - Australia
TA  - Clin Exp Ophthalmol
JT  - Clinical & experimental ophthalmology
JID - 100896531
SB  - IM
MH  - *Clinical Trials as Topic
MH  - Dependovirus/*genetics
MH  - Eye Diseases/genetics/*therapy
MH  - Eye Diseases, Hereditary/genetics/*therapy
MH  - Forecasting
MH  - *Genetic Therapy
MH  - Genetic Vectors/*genetics
MH  - Humans
MH  - Molecular Biology
MH  - Safety Management
OTO - NOTNLM
OT  - age-related macular degeneration
OT  - drug trial
OT  - gene expression
OT  - gene therapy
OT  - molecular genetics
EDAT- 2018/10/23 06:00
MHDA- 2020/06/06 06:00
CRDT- 2018/10/23 06:00
PHST- 2018/09/16 00:00 [received]
PHST- 2018/10/04 00:00 [revised]
PHST- 2018/10/15 00:00 [accepted]
PHST- 2018/10/23 06:00 [pubmed]
PHST- 2020/06/06 06:00 [medline]
PHST- 2018/10/23 06:00 [entrez]
AID - 10.1111/ceo.13416 [doi]
PST - ppublish
SO  - Clin Exp Ophthalmol. 2019 May;47(4):521-536. doi: 10.1111/ceo.13416. Epub 2019 
      Jan 2.