ReviewVision research2008
Barriers for retinal gene therapy: separating fact from fiction.
Review in Vision research, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
18 citing papers in PubMed, 42 citations in OpenAlex.
- Trial
- Peripheral monocytes and neutrophils promote photoreceptor cell death in an experimental retinal detachment model.Cell death & disease · 2023Article
- RIPK necrotic cell death pathway in both donor photoreceptor and host immune cells synergize to affect photoreceptor graft survival.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2023Article
- USH2A Gene Editing Using the CRISPR System.Molecular therapy. Nucleic acids · 2017Article
- Gene therapy of inherited retinal degenerations: prospects and challenges.Human gene therapy · 2015Review
- Gene Therapy of ABCA4-Associated Diseases.Cold Spring Harbor perspectives in medicine · 2015Review
- Lentiviral vectors as tools to understand central nervous system biology in mammalian model organisms.Frontiers in molecular neuroscience · 2015Review
- Vector platforms for gene therapy of inherited retinopathies.Progress in retinal and eye research · 2014Review
- Retinal transduction profiles by high-capacity viral vectors.Gene therapy · 2014Article
- Sustained gene expression in the retina by improved episomal vectors.Tissue engineering. Part A · 2014Article
- Gene replacement therapy for retinal CNG channelopathies.Molecular genetics and genomics : MGG · 2013Review
- Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients.Human gene therapy · 2012Review
- Gene therapy for retinal ganglion cell neuroprotection in glaucoma.Gene therapy · 2012Review
- Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model.Proceedings of the National Academy of Sciences of the United States of America · 2011Article
- Helper-Dependent Adenoviral Vectors.Journal of genetic syndromes & gene therapy · 2011Article
- Subretinal gene delivery using helper-dependent adenoviral vectors.Cell & bioscience · 2011Article
- Gene therapy with helper-dependent adenoviral vectors: current advances and future perspectives.Viruses · 2010Article
- A poly(ethylene) glycolylated peptide for ocular delivery compacts DNA into nanoparticles for gene delivery to post-mitotic tissues in vivo.The journal of gene medicine · 2010Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
The majority of recent preclinical gene therapy studies targeting the retina have used adeno-associated virus (AAV) as the gene transfer vector. However, AAV has several limitations including the ability to generate innate inflammatory responses, the ability to cause insertional mutagenesis at a frequency of up to 56% in some tissues and a limited cloning capacity of 4.8Kb. Furthermore, AAV is known to generate limiting immune responses in humans despite the absence of similar immune responses in preclinical canine and murine studies. Three clinical trials to treat Leber's congenital amaurosis using AAV are under way. A clinical trial to treat Stargardt's using lentivirus vectors has also been recently announced. However, very limited evidence currently exists that lentivirus vectors can efficiently transduce photoreceptor cells. In contrast, very few preclinical ocular gene therapy studies have utilized adenovirus as the gene therapy vector. Nonetheless, the only two ocular gene therapy clinical trials performed to date have each used adenovirus as the vector and more significantly, in these published trials there has been no observed serious adverse event. These trials appear to be poised for Phase II/III status. Activation of cytotoxic T lymphocytes limits duration of transgene expression in the retina from first generation adenovirus vectors. However, an advanced class of adenovirus vectors referred to as Helper-dependent Adenovirus (Hd-Ad) have recently been shown to be capable of expressing transgenes in ocular tissues for more than one year. Hd-Ad vectors have many properties that potentially warrant their inclusion in the retinal gene therapy toolbox for the treatment of retinal degenerative diseases.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.