ArticleHuman gene therapy2019
Somatic Gene Editing of
Open access · greenAbstract read
In one paragraph
Article in Human gene therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 52 papers.
0numbers the graph read from it
0cells of the map it votes in
52citing papers in PubMed
3.5field-weighted citation impact, top 6% of its field
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
52 citing papers in PubMed, 78 citations in OpenAlex.
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- Non-clinical safety considerations on genome editing using the CRISPR/Cas system.Genes & diseases · 2026Review
- Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Safety and efficacy of CRISPR-mediated genome ablation of VEGFA as a treatment for choroidal neovascularization in nonhuman primate eyes.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- CRISPR/Cas9 gene therapy increases the risk of tumorigenesis in the mouse model of hereditary tyrosinemia type I.JHEP reports : innovation in hepatology · 2025Article
- CRISPR targeting of mmu-miR-21a through a single adeno-associated virus vector prolongs survival of glioblastoma-bearing mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Next generation therapeutics for retinal neurodegenerative diseases.Journal of controlled release : official journal of the Controlled Release Society · 2024Review
- An Update on the Application of CRISPR Technology in Clinical Practice.Molecular biotechnology · 2024Review
- Engineered AAV2.7m8 Serotype Shows Significantly Higher Transduction Efficiency of ARPE-19 and HEK293 Cell Lines Compared to AAV5, AAV8 and AAV9 Serotypes.Pharmaceutics · 2024Article
- A natural history study of autosomal dominant GUCY2D-associated cone-rod dystrophy.Documenta ophthalmologica. Advances in ophthalmology · 2023Article
- Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6.Molecular therapy. Methods & clinical development · 2023Article
- Translatability barriers between preclinical and clinical trials of AAV gene therapy in inherited retinal diseases.Vision research · 2023Review
- Article
- Recent advances in therapeutic CRISPR-Cas9 genome editing: mechanisms and applications.Molecular biomedicine · 2023Review
- Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Gene therapy for inherited retinal diseases: exploiting new tools in genome editing and nanotechnology.Frontiers in ophthalmology · 2023Review
- Advances in CRISPR/Cas-Based Strategies on Zoonosis.Transboundary and emerging diseases · 2023Review
- Population-wide gene disruption in the murine lung epithelium via AAV-mediated delivery of CRISPR-Cas9 components.Molecular therapy. Methods & clinical development · 2022Article
- Article
- CRISPR/Cas systems usher in a new era of disease treatment and diagnosis.Molecular biomedicine · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors at 3 institutions in 1 country.
K Tyler McCullough1 Department of Ophthalmology, University of Florida, Gainesville, Florida.
Sanford L Boye1 Department of Ophthalmology, University of Florida, Gainesville, Florida.
Diego Fajardo1 Department of Ophthalmology, University of Florida, Gainesville, Florida.
Kaitlyn Calabro1 Department of Ophthalmology, University of Florida, Gainesville, Florida.
James J Peterson1 Department of Ophthalmology, University of Florida, Gainesville, Florida.
Christianne E Strang2 Department of Ophthalmology, University of Alabama at Birmingham, Birmingham, Alabama.
Dibyendu Chakraborty2 Department of Ophthalmology, University of Alabama at Birmingham, Birmingham, Alabama.
Sebastian Gloskowski3 Editas Medicine, Cambridge, Massachusetts.
Scott Haskett3 Editas Medicine, Cambridge, Massachusetts.
Steven Samuelsson3 Editas Medicine, Cambridge, Massachusetts.
Haiyan Jiang3 Editas Medicine, Cambridge, Massachusetts.
C Douglas Witherspoon2 Department of Ophthalmology, University of Alabama at Birmingham, Birmingham, Alabama.
Paul D Gamlin2 Department of Ophthalmology, University of Alabama at Birmingham, Birmingham, Alabama.
Morgan L Maeder3 Editas Medicine, Cambridge, Massachusetts.
Shannon E Boye1 Department of Ophthalmology, University of Florida, Gainesville, Florida.
University of Florida · USUniversity of Alabama at Birmingham · USEditas Medicine (United States) · US
Funding
Visual Phenotyping CoreP30EY003039 · NEI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI Paul Douglas Gamlin · 1985 to 2026
$16.9MEngineering AAV for safe and efficient gene delivery to the human retinaR01EY024280 · NEI · UNIVERSITY OF FLORIDA · PI Shannon Elizabeth Boye · 2014 to 2026
$7.5MIntrinsically photosensitive retinal ganglion cells and their central projectionsR01EY025555 · NEI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI DO, MICHAEL TRI HOANG, GAMLIN, PAUL DOUGLAS · 2016 to 2020
$3.9MRESEARCH TRAINING IN VISION SCIENCET32EY007132 · NEI · UNIVERSITY OF FLORIDA · PI W CLAY SMITH · 1991 to 2026
$3.8MNEI NIH HHS P30 EY003039NEI NIH HHS R01 EY024280NEI NIH HHS R01 EY025555NEI NIH HHS T32 EY007132
Abstract
Mutations in
Indexed as
CRISPR-Cas SystemsGene EditingAnimalsBase SequenceDependovirusElectroretinographyGenes, ReporterGenetic VectorsGuanylate CyclaseMacacaMiceMice, KnockoutMolecular ImagingPromoter Regions, GeneticReceptors, Cell SurfaceRetinaGuanylate CyclaseGucy2d protein, mouseReceptors, Cell SurfaceRNA, Guide, CRISPR-Cas SystemsAAVcone rod dystrophyCRISPR/Cas9gene editingGUCY2Dretina
Identifiers
What OpenQuestion holds
Textmetadata
Read underepoch 390
Registered trials
None linked
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.