OOIR: Observatory of International Research

Papers

(The median citation count of Gene Therapy is 3. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2021-08-01 to 2025-08-01.)

Article	Citations
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors	98
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice	59
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings	58
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury	46
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia	45
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model	39
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	37
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system	35
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy	34
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials	34
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry	33
Implications of maternal-fetal health on perinatal stem cell banking	31
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2	31
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa	27
Correction: The disparate burden of infectious diseases	27
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene	24
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production	23
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis	23
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer	22
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	21
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice	20
Unlocking patient access to gene therapy: five key practices	19
Injection pressure levels for creating blebs during subretinal gene therapy	19
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1	17
Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea	17

Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression	17
hInGeTox: a human-based in vitro platform to evaluate lentivirus/host interactions that contribute to genotoxicity	17
Analytical characterization of full, intermediate, and empty AAV capsids	17
Correction: Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)	16
Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application	15
Epigenetic repression of gonadotropin gene expression via a GnRH-mediated DNA delivery system	15
Prevalence of AAV2.5 neutralizing antibodies in synovial fluid and serum of patients with osteoarthritis	15
Approval and therapeutic value of gene therapies in the US and Europe	15
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent	15
An ectopic enhancer restores CFTR expression through de novo chromatin looping	14
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing	14
A PCR-amplified transgene fragment flanked by a single copy of a truncated inverted terminal repeat for recombinant adeno-associated virus production prevents unnecessary plasmid DNA packaging	14
Retraction Note: Optimization of oncolytic effect of Newcastle disease virus Clone30 by selecting sensitive tumor host and constructing more oncolytic viruses	14
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia	13
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques	13
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice	13
A short hepatitis C virus NS5A peptide expression by AAV vector modulates human T cell activation and reduces vector immunogenicity	13
Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve	12
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality	12
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver	11
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage	11
Ghrelin mediated cardioprotection using in vitro models of oxidative stress	11
Gene drives: an alternative approach to malaria control?	11
Correction: Antitumoral gene-based strategy involving nitric oxide synthase type III overexpression in hepatocellular carcinoma	11
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation	10
Correction: AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses	10
Gene therapy corrects the neurological deficits of mice with sialidosis	10
Correction: RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells	10
Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice	10
Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	10
CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model	10
Retraction Note: Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH1	9
Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells	9
Characterization of anti-AAV2 neutralizing antibody levels in sheep prior to and following intravitreal AAV2.7m8 injection	9
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study	9
Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential	9
Rational engineering of a functional CpG-free ITR for AAV gene therapy	9
A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies	8
Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes	8
Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications	8
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model	8
Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos	8
Correction of a knock-in mouse model of acrodysostosis with gene therapy using a rAAV9-CAG-human PRKAR1A vector	8
Exosomal PD-L1 confers chemoresistance and promotes tumorigenic properties in esophageal cancer cells via upregulating STAT3/miR-21	8
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy	8
Correction: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65	8
AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses	8
CCR1 enhances SUMOylation of DGCR8 by up-regulating ERK phosphorylation to promote spinal nerve ligation-induced neuropathic pain	8
Prime editing: therapeutic advances and mechanistic insights	8
Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)	8

AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis	8
Genes for bad backs	8
Acoustically targeted noninvasive gene therapy in large brain volumes	7
Optimized expression of alternative oxidase	7
The disparate burden of infectious diseases	7
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification	7
Targeting mAKAPβ expression as a therapeutic approach for ischemic cardiomyopathy	7
Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model	7
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement	7
Glyco-binding domain chimeric antigen receptors as a new option for cancer immunotherapy	7
Correction: Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors	6
Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy	6
Systemically delivered lipid nanoparticle-mRNA encoding lysosomal acid β-glucosidase restores the enzyme deficiency in a murine Gaucher disease model	6
PCRX-201, a novel IL-1Ra gene therapy treatment approach for low back pain resulting from intervertebral disc degeneration	6
Incomplete elimination of viral genomes is associated with chronic inflammation in nonhuman primate livers after AAV-mediated gene transfer	6
Splicing mutations in the CFTR gene as therapeutic targets	6
Administration and detection of a multi-target rAAV gene doping vector in horses using multiple matrices and molecular techniques	6
Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing	6
Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability	6
Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2 : a systematic review and meta-analysis	5
Successes and challenges in clinical gene therapy	5
Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo	5
Distributional comparison of different AAV vectors after unilateral cochlear administration	5
Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model	5
AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial	5
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy	5
Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation	5
Effect of nanoparticle-mediated delivery of SFRP4 siRNA for treating Dupuytren disease	5
Selective attachment of a microtubule interacting peptide to plasmid DNA via a triplex forming oligonucleotide for transfection improvement	4
Stable inhibition of choroidal neovascularization by adeno-associated virus 2/8-vectored bispecific molecules	4
Upper motor neurons are a target for gene therapy and UCHL1 is necessary and sufficient to improve cellular integrity of diseased upper motor neurons	4
Aptamer-targeted anti-miR RNA construct based on 3WJ as a new approach for the treatment of chronic kidney disease in an experimental model	4
Curing SMA: Are we there yet?	4
Friend or Foe(tal): challenges in development of a large animal model for pre-clinical fetal gene therapy	4
Thrombopoietin-based CAR-T cells demonstrate in vitro and in vivo cytotoxicity to MPL positive acute myelogenous leukemia and hematopoietic stem cells	4
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute	4
Ex vivo machine perfusion as a platform for lentiviral gene delivery in rat livers	4
A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice	4
Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9	4
AAV vector engineering for human aorta transduction: becoming a smooth operator	4
Placental nanoparticle-mediated IGF1 gene therapy corrects fetal growth restriction in a guinea pig model	4
The estimated annual financial impact of gene therapy in the United States	4
A multinational survey of potential participant perspectives on ocular gene therapy	3
Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system	3
Nitrous oxide enhances MR-guided focused ultrasound delivery of gene therapy to the murine hippocampus	3
Unveiling the sex bias: higher preexisting and neutralizing titers against AAV in females and implications for gene therapy	3
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model	3
The impact of heparin and direct thrombin inhibitors on cell-penetrating polymer siRNA transfection	3
Design, construction and in vivo functional assessment of a hinge truncated sFLT01	3
Effective viral-mediated lung gene therapy: is airway surface preparation necessary?	3
Unveiling molecular secrets: Analysis of stable lentiviral packaging cell lines enables identification of novel viral gene functions	3
Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia	3
Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets	3
Cost of gene therapy	3
Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	3
HIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival	3
Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice	3
Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells	3
Terminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail	3
Outer retinal transduction by AAV2-7m8 following intravitreal injection in a sheep model of CNGA3 achromatopsia	3
RNAi targeting heparin cofactor II promotes hemostasis in a canine model of acquired hemophilia A	3
Equitable access to cell and gene therapies in South Africa: opportunities and hurdles	3
BRD9 promotes the progression of gallbladder cancer via CST1 upregulation and interaction with FOXP1 through the PI3K/AKT pathway and represents a therapeutic target	3
Forced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution	3
Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy	3
hafoe: an interactive tool for the analysis of chimeric AAV libraries after random mutagenesis	3
Retraction Note: Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations	3
Spotlight on gene therapy in Germany	3
Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model	3
Precision medicine: toward restoring fat with gene therapy in inherited lipodystrophy	3
Retraction Note: Oncolysis of pancreatic tumour cells by a γ34.5-deleted HSV-1 does not rely upon Ras-activation, but on the PI 3-kinase pathway	3
CRISPR editing as a therapeutic strategy for Duchenne muscular dystrophy—anti-Cas9 immune response casts its shadow over safety and efficacy	3
Electroporation-mediated novel albumin-fused Flt3L DNA delivery promotes cDC1-associated anticancer immunity	3
Correction: Alternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active	3