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 2022-05-01 to 2026-05-01.)

Article	Citations
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia	146
Correction: PPARγ is essential for protection against nonalcoholic steatohepatitis	68
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	67
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings	60
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice	57
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors	51
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy	45
Codon changes challenge PCR-based gene doping detection	43
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials	35
Drinkable gene therapy foam for the treatment of constrictive esophageal carcinoma	32
First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study	31
Beneficial bystander-enhanced cryptic splice rescue of cardiac-type Fabry GLA IVS4+919G>A by adenine base editing in patient fibroblasts	28
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system	27
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2	27
The co-delivery of Programmed Death 1 ligands enhances and prolongs rAAV-mediated gene expression in pre-immunized mice	26
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery	26
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa	26
Implications of maternal-fetal health on perinatal stem cell banking	24
Correction: The disparate burden of infectious diseases	24
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production	23
Murine toxicology assessment of avgn7.2, a novel gene therapeutic for inclusion body myositis and other muscle wasting diseases	22
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	21
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis	21
hInGeTox: a human-based in vitro platform to evaluate lentivirus/host interactions that contribute to genotoxicity	20
Topical application of Cas9 ribonucleoproteins inhibits corneal neovascularization in a mouse model of alkali burn injury	20

Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression	19
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice	18
Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea	17
Unlocking patient access to gene therapy: five key practices	17
The poly-(HA-GMA) hydrogel carrying AAV8-sTβRII alleviates scar formation in mice skin wound healing by inhibiting fibrosis	17
Analytical characterization of full, intermediate, and empty AAV capsids	17
Approval and therapeutic value of gene therapies in the US and Europe	16
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent	15
Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application	15
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality	14
Retraction Note: Optimization of oncolytic effect of Newcastle disease virus Clone30 by selecting sensitive tumor host and constructing more oncolytic viruses	14
Seroprevalence of anti-AAV antibodies in a healthy adult Spanish population: findings from the SAAVIA study	13
Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve	13
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia	13
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice	13
An ectopic enhancer restores CFTR expression through de novo chromatin looping	13
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques	13
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver	12
Correction: Antitumoral gene-based strategy involving nitric oxide synthase type III overexpression in hepatocellular carcinoma	12
Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates	12
Vaccine elicitation of HIV broadly neutralizing antibodies from genome-edited B cells in non-human primates and derived lymphoid organoids	12
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage	12
Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda’s context	12
CRISPR-AuNP: physicochemical optimization of a gold nanoparticle platform for cost-effective and modular non-viral gene editing in HSPCs	12
Correction: Expression of anti-amyloid CARs in microglia promotes efficient and selective phagocytosis of Aβ1‒42	11
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation	11
Ghrelin mediated cardioprotection using in vitro models of oxidative stress	11
Gene drives: an alternative approach to malaria control?	11
Correction: RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells	11
Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	11
Dystrophin/mini-dystrophin expression analysis by immunoaffinity liquid chromatography–tandem mass spectrometry after gene therapy for DMD	10
CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model	10
Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice	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
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study	10
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model	9
AAV-based gene therapies for neovascular AMD	9
Retraction Note: Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH1	9
Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential	9
Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes	9
Characterization of anti-AAV2 neutralizing antibody levels in sheep prior to and following intravitreal AAV2.7m8 injection	9
Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications	9
AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis	9
Prime editing: therapeutic advances and mechanistic insights	8
Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos	8
Acoustically targeted noninvasive gene therapy in large brain volumes	8
Genes for bad backs	8
Correction: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65	8
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

Preclinical safety and biodistribution of SPVN06, a novel gene- and mutation-independent gene therapy for rod-cone dystrophies	7
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement	7
Optimized expression of alternative oxidase	7
Correction: Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates	7
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification	7
Codon-optimized human Smad7 gene therapy enhances skeletal muscle mass and function in a murine model of Duchenne muscular dystrophy	7
Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model	7
Lipid nanoparticle mediated base editing of the Q344X rhodopsin mutation associated with retinitis pigmentosa	6
Glyco-binding domain chimeric antigen receptors as a new option for cancer immunotherapy	6
Correction: Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors	6
Baboon endogenous retrovirus (ERV) envelope pseudotyped lentiviral vectors outperform human ERV lentivectors for transduction of T, B, NK and HSPCs	6
The disparate burden of infectious diseases	6
Gene therapy restores auditory function and rescues damaged inner hair cells in an aged Vglut3 knockout mouse model	6
Administration and detection of a multi-target rAAV gene doping vector in horses using multiple matrices and molecular techniques	6
Successes and challenges in clinical gene therapy	6
Gene editing for collagen disorders: current advances and future perspectives	6
Securitization as a means to pay for cell and gene therapies for orphan diseases: a simulation study	6
Splicing mutations in the CFTR gene as therapeutic targets	6
PCRX-201, a novel IL-1Ra gene therapy treatment approach for low back pain resulting from intervertebral disc degeneration	5
Systemically delivered lipid nanoparticle-mRNA encoding lysosomal acid β-glucosidase restores the enzyme deficiency in a murine Gaucher disease 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
Ex vivo machine perfusion as a platform for lentiviral gene delivery in rat livers	5
Placental nanoparticle-mediated IGF1 gene therapy corrects fetal growth restriction in a guinea pig model	5
AAV-mediated BDNF and GAS6 muscle delivery delays disease onset in SOD1G93A ALS mice	5
Incomplete elimination of viral genomes is associated with chronic inflammation in nonhuman primate livers after AAV-mediated gene transfer	5
Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2 : a systematic review and meta-analysis	5
Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model	5
AAV vector engineering for human aorta transduction: becoming a smooth operator	5
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
Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing	5
Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability	5
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute	5
Intranasal versus intravenous AAV delivery: A comparative analysis of brain-targeting efficiency and peripheral exposure in mice	5
Distributional comparison of different AAV vectors after unilateral cochlear administration	5
The estimated annual financial impact of gene therapy in the United States	4
A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice	4
Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	4
Precision rewriting of muscle genetics: therapeutic horizons of base and prime editing in skeletal muscle disorders	4
Nitrous oxide enhances MR-guided focused ultrasound delivery of gene therapy to the murine hippocampus	4
AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects	4
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
A multinational survey of potential participant perspectives on ocular gene therapy	4
Cost of gene therapy	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
Antimicrobial resistance and gene therapy: emerging molecular strategies for a global health threat	4
Forced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution	4
Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy	4
Electroporation-mediated novel albumin-fused Flt3L DNA delivery promotes cDC1-associated anticancer immunity	4
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
Correction: Alternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active	3
hafoe: an interactive tool for the analysis of chimeric AAV libraries after random mutagenesis	3
CRISPR/Cas9-mediated gene correction of Wilson disease H1069Q point mutation in patient-specific induced pluripotent stem cells	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
Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells	3
Therapeutic precision gene editing of cholesterol pathways as a gene therapy strategy for cardiovascular disease	3
HIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival	3
Unveiling the sex bias: higher preexisting and neutralizing titers against AAV in females and implications for gene therapy	3
RNAi targeting heparin cofactor II promotes hemostasis in a canine model of acquired hemophilia A	3
Retraction Note: Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations	3
Terminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail	3
Precision medicine: toward restoring fat with gene therapy in inherited lipodystrophy	3
Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia	3
Unveiling molecular secrets: Analysis of stable lentiviral packaging cell lines enables identification of novel viral gene functions	3
Retraction Note: Local administration of transcription factor decoy oligonucleotides to nuclear factor-κB prevents carrageenin-induced inflammation in rat hind paw	3
Design, construction and in vivo functional assessment of a hinge truncated sFLT01	3
The impact of heparin and direct thrombin inhibitors on cell-penetrating polymer siRNA transfection	3
Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model	3