OOIR: Observatory of International Research

Papers

(The TQCC of Gene Therapy is 8. 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
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry	34
Codon changes challenge PCR-based gene doping detection	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
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: The disparate burden of infectious diseases	23
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery	23
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
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	21
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
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice	20

hInGeTox: a human-based in vitro platform to evaluate lentivirus/host interactions that contribute to genotoxicity	19
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
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis	18
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
Prevalence of AAV2.5 neutralizing antibodies in synovial fluid and serum of patients with osteoarthritis	17
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
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
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
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice	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
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
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
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
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
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
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
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
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
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
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