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 2021-09-01 to 2025-09-01.)

Article	Citations
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors	103
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	48
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury	47
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia	43
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model	39
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy	35
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	35
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	34
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system	32
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	29
Correction: The disparate burden of infectious diseases	28
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa	25
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production	23
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene	23
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice	21
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis	21
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	21
hInGeTox: a human-based in vitro platform to evaluate lentivirus/host interactions that contribute to genotoxicity	21
Unlocking patient access to gene therapy: five key practices	19
Analytical characterization of full, intermediate, and empty AAV capsids	18
Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression	18
Correction: Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)	17

Injection pressure levels for creating blebs during subretinal gene therapy	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
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1	17
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent	16
Epigenetic repression of gonadotropin gene expression via a GnRH-mediated DNA delivery system	16
Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application	15
Retraction Note: Optimization of oncolytic effect of Newcastle disease virus Clone30 by selecting sensitive tumor host and constructing more oncolytic viruses	15
An ectopic enhancer restores CFTR expression through de novo chromatin looping	15
Approval and therapeutic value of gene therapies in the US and Europe	15
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	15
Prevalence of AAV2.5 neutralizing antibodies in synovial fluid and serum of patients with osteoarthritis	15
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing	15
A short hepatitis C virus NS5A peptide expression by AAV vector modulates human T cell activation and reduces vector immunogenicity	14
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques	14
Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve	14
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice	13
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver	13
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality	13
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia	13
Correction: RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells	11
Ghrelin mediated cardioprotection using in vitro models of oxidative stress	11
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage	11
Correction: Antitumoral gene-based strategy involving nitric oxide synthase type III overexpression in hepatocellular carcinoma	11
Gene drives: an alternative approach to malaria control?	11
Correction: Expression of anti-amyloid CARs in microglia promotes efficient and selective phagocytosis of Aβ1‒42	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
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation	10
Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates	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
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
Dystrophin/mini-dystrophin expression analysis by immunoaffinity liquid chromatography–tandem mass spectrometry after gene therapy for DMD	9
Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells	9
Rational engineering of a functional CpG-free ITR for AAV gene therapy	9
Exosomal PD-L1 confers chemoresistance and promotes tumorigenic properties in esophageal cancer cells via upregulating STAT3/miR-21	8
AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis	8
Correction: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65	8
Retraction Note: Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH1	8
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy	8
Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications	8
Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes	8
Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)	8
Prime editing: therapeutic advances and mechanistic insights	8
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model	8