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 2022-01-01 to 2026-01-01.)

Article	Citations
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice	123
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model	62
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors	55
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury	53
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia	49
Correction: PPARγ is essential for protection against nonalcoholic steatohepatitis	44
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings	44
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	40
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy	39
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system	37
Codon changes challenge PCR-based gene doping detection	34
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry	34
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials	30
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa	27
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production	24
Correction: The disparate burden of infectious diseases	23
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery	23
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene	23
Murine toxicology assessment of avgn7.2, a novel gene therapeutic for inclusion body myositis and other muscle wasting diseases	23
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	21
Implications of maternal-fetal health on perinatal stem cell banking	21
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2	21
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice	20
Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression	20
Unlocking patient access to gene therapy: five key practices	20

hInGeTox: a human-based in vitro platform to evaluate lentivirus/host interactions that contribute to genotoxicity	19
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis	18
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1	18
Analytical characterization of full, intermediate, and empty AAV capsids	18
Prevalence of AAV2.5 neutralizing antibodies in synovial fluid and serum of patients with osteoarthritis	17
Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application	17
Epigenetic repression of gonadotropin gene expression via a GnRH-mediated DNA delivery system	17
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent	17
Approval and therapeutic value of gene therapies in the US and Europe	16
An ectopic enhancer restores CFTR expression through de novo chromatin looping	16
Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea	16
Retraction Note: Optimization of oncolytic effect of Newcastle disease virus Clone30 by selecting sensitive tumor host and constructing more oncolytic viruses	16
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice	15
Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve	15
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia	15
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality	15
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver	13
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques	13
Ghrelin mediated cardioprotection using in vitro models of oxidative stress	12
Correction: Antitumoral gene-based strategy involving nitric oxide synthase type III overexpression in hepatocellular carcinoma	12
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage	12
Gene drives: an alternative approach to malaria control?	12
Correction: Expression of anti-amyloid CARs in microglia promotes efficient and selective phagocytosis of Aβ1‒42	12
Correction: RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells	12
Correction: AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses	11
Dystrophin/mini-dystrophin expression analysis by immunoaffinity liquid chromatography–tandem mass spectrometry after gene therapy for DMD	11
Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates	11
Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	11
Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice	11
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation	11
CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model	11
Retraction Note: Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH1	10
Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells	10
Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes	10
Gene therapy corrects the neurological deficits of mice with sialidosis	10
Characterization of anti-AAV2 neutralizing antibody levels in sheep prior to and following intravitreal AAV2.7m8 injection	10
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study	10
Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential	9
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model	9
Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications	9
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy	9
AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis	9
Exosomal PD-L1 confers chemoresistance and promotes tumorigenic properties in esophageal cancer cells via upregulating STAT3/miR-21	9
Prime editing: therapeutic advances and mechanistic insights	9
Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos	8
AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses	8
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification	8
Codon-optimized human Smad7 gene therapy enhances skeletal muscle mass and function in a murine model of Duchenne muscular dystrophy	8
Correction: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65	8
Genes for bad backs	8

Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement	8
Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model	7
Glyco-binding domain chimeric antigen receptors as a new option for cancer immunotherapy	7
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	7
Acoustically targeted noninvasive gene therapy in large brain volumes	7
Targeting mAKAPβ expression as a therapeutic approach for ischemic cardiomyopathy	7
Administration and detection of a multi-target rAAV gene doping vector in horses using multiple matrices and molecular techniques	7
Correction: Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates	7
Gene therapy restores auditory function and rescues damaged inner hair cells in an aged Vglut3 knockout mouse model	7
Optimized expression of alternative oxidase	7
Gene editing for collagen disorders: current advances and future perspectives	6
Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability	6
Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy	6
Correction: Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors	6
Splicing mutations in the CFTR gene as therapeutic targets	6
Preclinical safety and biodistribution of SPVN06, a novel gene- and mutation-independent gene therapy for rod-cone dystrophies	6
Incomplete elimination of viral genomes is associated with chronic inflammation in nonhuman primate livers after AAV-mediated gene transfer	6
Distributional comparison of different AAV vectors after unilateral cochlear administration	6
Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2 : a systematic review and meta-analysis	6
The disparate burden of infectious diseases	6
Lipid nanoparticle mediated base editing of the Q344X rhodopsin mutation associated with retinitis pigmentosa	6
Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing	6
PCRX-201, a novel IL-1Ra gene therapy treatment approach for low back pain resulting from intervertebral disc degeneration	6
Systemically delivered lipid nanoparticle-mRNA encoding lysosomal acid β-glucosidase restores the enzyme deficiency in a murine Gaucher disease model	6
AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial	5
Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo	5
Ex vivo machine perfusion as a platform for lentiviral gene delivery in rat livers	5
AAV vector engineering for human aorta transduction: becoming a smooth operator	5
Successes and challenges in clinical gene therapy	5
AAV-mediated BDNF and GAS6 muscle delivery delays disease onset in SOD1G93A ALS mice	5
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute	5
Placental nanoparticle-mediated IGF1 gene therapy corrects fetal growth restriction in a guinea pig model	5
Effect of nanoparticle-mediated delivery of SFRP4 siRNA for treating Dupuytren disease	5
Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model	5
Aptamer-targeted anti-miR RNA construct based on 3WJ as a new approach for the treatment of chronic kidney disease in an experimental model	5
Electroporation-mediated novel albumin-fused Flt3L DNA delivery promotes cDC1-associated anticancer immunity	4
The estimated annual financial impact of gene therapy in the United States	4
Friend or Foe(tal): challenges in development of a large animal model for pre-clinical fetal gene therapy	4
Stable inhibition of choroidal neovascularization by adeno-associated virus 2/8-vectored bispecific molecules	4
Precision rewriting of muscle genetics: therapeutic horizons of base and prime editing in skeletal muscle disorders	4
A multinational survey of potential participant perspectives on ocular gene therapy	4
Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia	4
Selective attachment of a microtubule interacting peptide to plasmid DNA via a triplex forming oligonucleotide for transfection improvement	4
Curing SMA: Are we there yet?	4
Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9	4
Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	4
Nitrous oxide enhances MR-guided focused ultrasound delivery of gene therapy to the murine hippocampus	4
Correction: Alternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active	4
Forced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution	4
Intranasal versus intravenous AAV delivery: A comparative analysis of brain-targeting efficiency and peripheral exposure in mice	4
AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects	4
A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice	4
Cost of gene therapy	4
Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy	4
hafoe: an interactive tool for the analysis of chimeric AAV libraries after random mutagenesis	3
CRISPR editing as a therapeutic strategy for Duchenne muscular dystrophy—anti-Cas9 immune response casts its shadow over safety and efficacy	3
Early life safety profiling of gene therapy for spinal muscular atrophy	3
Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells	3
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model	3
Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model	3
Effective viral-mediated lung gene therapy: is airway surface preparation necessary?	3
RNAi targeting heparin cofactor II promotes hemostasis in a canine model of acquired hemophilia A	3
Design, construction and in vivo functional assessment of a hinge truncated sFLT01	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
Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease	3
Precision medicine: toward restoring fat with gene therapy in inherited lipodystrophy	3
Therapeutic precision gene editing of cholesterol pathways as a gene therapy strategy for cardiovascular disease	3
Retraction Note: Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations	3
HIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival	3
Unveiling molecular secrets: Analysis of stable lentiviral packaging cell lines enables identification of novel viral gene functions	3
Equitable access to cell and gene therapies in South Africa: opportunities and hurdles	3
The impact of heparin and direct thrombin inhibitors on cell-penetrating polymer siRNA transfection	3
Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice	3
AAV-mediated MUC5AC siRNA delivery to prevent mucociliary dysfunction in asthma	3
Terminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail	3
Unveiling the sex bias: higher preexisting and neutralizing titers against AAV in females and implications for gene therapy	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