ReviewMolecular therapy : the journal of the American Society of Gene Therapy2021
Genome editing in large animal models.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 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
28 citing papers in PubMed, 41 citations in OpenAlex.
- MnOMaterials today. Bio · 2026Article
- Neuroimmune Regulation in Posttraumatic Bone Repair: From Inflammatory Transition to Neurovascular Coupling and Functional Recovery.Journal of cellular physiology · 2026Review
- Towards clinical translation of nanomedicines: Formulation scale-up and model systems.Advanced drug delivery reviews · 2026Review
- Retinoic Acid Signaling in Male Reproductive Biology: From Germ Cell Regulation to Contraceptive Innovation Within a One Health Framework.Animals : an open access journal from MDPI · 2026Review
- Animal models of benign airway stenosis: Advances in construction techniques, evaluation systems, and perspectives.Animal models and experimental medicine · 2026Review
- Animal models in human surgery and implant innovation: ethical considerations and scientific advancements.Annals of medicine and surgery (2012) · 2025Article
- Effects ofAnimals : an open access journal from MDPI · 2025Article
- Article
- CRISPR Technology and Its Emerging Applications.Genomics, proteomics & bioinformatics · 2025Review
- Efficient generation of germline chimeras in a non-rodent species using rabbit induced pluripotent stem cells.Nature communications · 2025Article
- Bibliometric and visual analysis of miRNAs in heart diseases from 2004 to 2023.Frontiers in cardiovascular medicine · 2025Article
- Genetic engineering and the eye.Eye (London, England) · 2025Review
- Protein is expressed in all major organs after intravenous infusion of mRNA-lipid nanoparticles in swine.Molecular therapy. Methods & clinical development · 2024Article
- CRISPR technology in human diseases.MedComm · 2024Review
- Engineered cytosine base editor enabling broad-scope and high-fidelity gene editing in Streptomyces.Nature communications · 2024Article
- siRNA-mediated reduction of a circulating protein in swine using lipid nanoparticles.Molecular therapy. Methods & clinical development · 2024Article
- Impact of CRISPR/HDR editing versus lentiviral transduction on long-term engraftment and clonal dynamics of HSPCs in rhesus macaques.Cell stem cell · 2024Article
- Advances in protein subunit vaccines against H1N1/09 influenza.Frontiers in immunology · 2024Review
- The progress of induced pluripotent stem cells derived from pigs: a mini review of recent advances.Frontiers in cell and developmental biology · 2024Review
- Gene editing in small and large animals for translational medicine: a review.Animal reproduction · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 3 institutions in 2 countries.
Funding
Abstract
Although genome editing technologies have the potential to revolutionize the way we treat human diseases, barriers to successful clinical implementation remain. Increasingly, preclinical large animal models are being used to overcome these barriers. In particular, the immunogenicity and long-term safety of novel gene editing therapeutics must be evaluated rigorously. However, short-lived small animal models, such as mice and rats, cannot address secondary pathologies that may arise years after a gene editing treatment. Likewise, immunodeficient mouse models by definition lack the ability to quantify the host immune response to a novel transgene or gene-edited locus. Large animal models, including dogs, pigs, and non-human primates (NHPs), bear greater resemblance to human anatomy, immunology, and lifespan and can be studied over longer timescales with clinical dosing regimens that are more relevant to humans. These models allow for larger scale and repeated blood and tissue sampling, enabling greater depth of study and focus on rare cellular subsets. Here, we review current progress in the development and evaluation of novel genome editing therapies in large animal models, focusing on applications in human immunodeficiency virus 1 (HIV-1) infection, cancer, and genetic diseases including hemoglobinopathies, Duchenne muscular dystrophy (DMD), hypercholesterolemia, and inherited retinal 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.