PMID- 37878215
OWN - NLM
STAT- MEDLINE
DCOM- 20240117
LR  - 20240122
IS  - 1179-190X (Electronic)
IS  - 1173-8804 (Print)
IS  - 1173-8804 (Linking)
VI  - 38
IP  - 1
DP  - 2024 Jan
TI  - AAV-Based Strategies for Treatment of Retinal and Choroidal Vascular Diseases: 
      Advances in Age-Related Macular Degeneration and Diabetic Retinopathy Therapies.
PG  - 73-93
LID - 10.1007/s40259-023-00629-y [doi]
AB  - Age-related macular degeneration (AMD) and diabetic retinopathy (DR) are vascular 
      diseases with high prevalence, ranking among the leading causes of blindness and 
      vision loss worldwide. Despite being effective, current treatments for AMD and DR 
      are burdensome for patients and clinicians, resulting in suboptimal compliance 
      and real risk of vision loss. Thus, there is an unmet need for long-lasting 
      alternatives with improved safety and efficacy. Adeno-associated virus (AAV) is 
      the leading vector for ocular gene delivery, given its ability to enable 
      long-term expression while eliciting relatively mild immune responses. Progress 
      has been made in AAV-based gene therapies for not only inherited retinal diseases 
      but also acquired conditions with preclinical and clinical studies of AMD and DR 
      showing promising results. These studies have explored several pathways involved 
      in the disease pathogenesis, as well as different strategies to optimise gene 
      delivery. These include engineered capsids with enhanced tropism to particular 
      cell types, and expression cassettes incorporating elements for a targeted and 
      controlled expression. Multiple-acting constructs have also been investigated, in 
      addition to gene silencing and editing. Here, we provide an overview of 
      strategies employing AAV-mediated gene delivery to treat AMD and DR. We discuss 
      preclinical efficacy studies and present the latest data from clinical trials for 
      both diseases.
CI  - (c) 2023. The Author(s).
FAU - Castro, Brenda F M
AU  - Castro BFM
AUID- ORCID: 0000-0002-3604-8757
AD  - LVF Ophthalmology Research Centre, Translational Research Institute, Brisbane, 
      QLD, 4102, Australia. b.castro@uq.edu.au.
AD  - Greenslopes Clinical School, University of Queensland School of Medicine, 
      Brisbane, QLD, Australia. b.castro@uq.edu.au.
FAU - Steel, Jason C
AU  - Steel JC
AUID- ORCID: 0000-0003-3608-7542
AD  - LVF Ophthalmology Research Centre, Translational Research Institute, Brisbane, 
      QLD, 4102, Australia.
AD  - Greenslopes Clinical School, University of Queensland School of Medicine, 
      Brisbane, QLD, Australia.
AD  - School of Health, Medical and Applied Sciences, Central Queensland University, 
      Rockhampton, QLD, Australia.
FAU - Layton, Christopher J
AU  - Layton CJ
AUID- ORCID: 0000-0001-6933-9991
AD  - LVF Ophthalmology Research Centre, Translational Research Institute, Brisbane, 
      QLD, 4102, Australia. c.layton@uq.edu.au.
AD  - Greenslopes Clinical School, University of Queensland School of Medicine, 
      Brisbane, QLD, Australia. c.layton@uq.edu.au.
AD  - School of Health, Medical and Applied Sciences, Central Queensland University, 
      Rockhampton, QLD, Australia. c.layton@uq.edu.au.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20231025
PL  - New Zealand
TA  - BioDrugs
JT  - BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy
JID - 9705305
SB  - IM
MH  - Humans
MH  - *Diabetic Retinopathy/therapy/drug therapy
MH  - Dependovirus/genetics
MH  - *Choroidal Neovascularization/drug therapy/genetics
MH  - *Macular Degeneration/therapy/drug therapy
MH  - Gene Transfer Techniques
MH  - *Diabetes Mellitus
PMC - PMC10789843
COIS- Christopher J. Layton owns shares in Ocugene, a gene therapy company. Jason C. 
      Steel has been a consultant for Ocugene and owns intellectual property in breast 
      cancer gene therapy. Brenda F. M. Castro has no relevant financial or 
      non-financial interests to disclose.
EDAT- 2023/10/25 12:42
MHDA- 2024/01/17 06:42
PMCR- 2023/10/25
CRDT- 2023/10/25 11:12
PHST- 2023/09/20 00:00 [accepted]
PHST- 2024/01/17 06:42 [medline]
PHST- 2023/10/25 12:42 [pubmed]
PHST- 2023/10/25 11:12 [entrez]
PHST- 2023/10/25 00:00 [pmc-release]
AID - 10.1007/s40259-023-00629-y [pii]
AID - 629 [pii]
AID - 10.1007/s40259-023-00629-y [doi]
PST - ppublish
SO  - BioDrugs. 2024 Jan;38(1):73-93. doi: 10.1007/s40259-023-00629-y. Epub 2023 Oct 
      25.