ReviewProtein & cell2011
Gene therapy: light is finally in the tunnel.
Review in Protein & cell, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 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
11 citing papers in PubMed, 21 citations in OpenAlex.
- SynBioNanoDesign: pioneering targeted drug delivery with engineered nanomaterials.Journal of nanobiotechnology · 2025Review
- Direct Adeno-associated Viruses Injection of Murine Adipose Tissue.Bio-protocol · 2023Article
- Recent Advancements in Gene Therapy for Hereditary Retinal Dystrophies.Turkish journal of ophthalmology · 2017Review
- Targeting Visceral Fat by Intraperitoneal Delivery of Novel AAV Serotype Vector Restricting Off-Target Transduction in Liver.Molecular therapy. Methods & clinical development · 2017Article
- Leber's congenital amaurosis and the role of gene therapy in congenital retinal disorders.International journal of ophthalmology · 2017Review
- Testing gene therapy vectors in human primary nasal epithelial cultures.Molecular therapy. Methods & clinical development · 2015Article
- Lung gene therapy-How to capture illumination from the light already present in the tunnel.Genes & diseases · 2014Article
- Targeting adipose tissue via systemic gene therapy.Gene therapy · 2014Article
- Therapeutic potential of small molecules and engineered proteins.Handbook of experimental pharmacology · 2014Article
- A "so cilia" network: cilia proteins start "social" networking.The Journal of clinical investigation · 2012Article
- Retinal photodamage mediated by all-trans-retinal.Photochemistry and photobiologyReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
Abstract
After two decades of ups and downs, gene therapy has recently achieved a milestone in treating patients with Leber's congenital amaurosis (LCA). LCA is a group of inherited blinding diseases with retinal degeneration and severe vision loss in early infancy. Mutations in several genes, including RPE65, cause the disease. Using adeno-associated virus as a vector, three independent teams of investigators have recently shown that RPE65 can be delivered to retinal pigment epithelial cells of LCA patients by subretinal injections resulting in clinical benefits without side effects. However, considering the whole field of gene therapy, there are still major obstacles to clinical applications for other diseases. These obstacles include innate and immune barriers to vector delivery, toxicity of vectors and the lack of sustained therapeutic gene expression. Therefore, new strategies are needed to overcome these hurdles for achieving safe and effective gene therapy. In this article, we shall review the major advancements over the past two decades and, using lung gene therapy as an example, discuss the current obstacles and possible solutions to provide a roadmap for future gene therapy research.
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.