OOIR: Observatory of International Research

Papers

(The TQCC of Gene Therapy is 7. 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 2022-06-01 to 2026-06-01.)

Article	Citations
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia	151
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice	72
Correction: PPARγ is essential for protection against nonalcoholic steatohepatitis	68
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings	65
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors	60
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	43
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy	35
Beneficial bystander-enhanced cryptic splice rescue of cardiac-type Fabry GLA IVS4+919G>A by adenine base editing in patient fibroblasts	33
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system	31
Drinkable gene therapy foam for the treatment of constrictive esophageal carcinoma	30
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials	28
Codon changes challenge PCR-based gene doping detection	28
First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study	27
The co-delivery of Programmed Death 1 ligands enhances and prolongs rAAV-mediated gene expression in pre-immunized mice	26
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2	26
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa	25
Implications of maternal-fetal health on perinatal stem cell banking	24
Correction: The disparate burden of infectious diseases	24
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery	23
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	22
Murine toxicology assessment of avgn7.2, a novel gene therapeutic for inclusion body myositis and other muscle wasting diseases	22
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis	22
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production	22
hInGeTox: a human-based in vitro platform to evaluate lentivirus/host interactions that contribute to genotoxicity	21
Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression	19

Analytical characterization of full, intermediate, and empty AAV capsids	17
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice	17
Topical application of Cas9 ribonucleoproteins inhibits corneal neovascularization in a mouse model of alkali burn injury	17
Characterization of CAR-T cell factors that contribute to myeloid cell activation	17
Unlocking patient access to gene therapy: five key practices	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
Approval and therapeutic value of gene therapies in the US and Europe	15
Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application	14
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent	14
An ectopic enhancer restores CFTR expression through de novo chromatin looping	13
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality	13
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques	13
Seroprevalence of anti-AAV antibodies in a healthy adult Spanish population: findings from the SAAVIA study	13
The poly-(HA-GMA) hydrogel carrying AAV8-sTβRII alleviates scar formation in mice skin wound healing by inhibiting fibrosis	13
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia	13
Retraction Note: Optimization of oncolytic effect of Newcastle disease virus Clone30 by selecting sensitive tumor host and constructing more oncolytic viruses	13
Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda’s context	12
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice	12
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver	12
Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve	12
CRISPR-AuNP: physicochemical optimization of a gold nanoparticle platform for cost-effective and modular non-viral gene editing in HSPCs	11
Correction: RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells	11
Vaccine elicitation of HIV broadly neutralizing antibodies from genome-edited B cells in non-human primates and derived lymphoid organoids	11
Correction: Expression of anti-amyloid CARs in microglia promotes efficient and selective phagocytosis of Aβ1‒42	11
Correction: Antitumoral gene-based strategy involving nitric oxide synthase type III overexpression in hepatocellular carcinoma	11
Ghrelin mediated cardioprotection using in vitro models of oxidative stress	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
Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates	10
Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	10
Gene drives: an alternative approach to malaria control?	10
Dystrophin/mini-dystrophin expression analysis by immunoaffinity liquid chromatography–tandem mass spectrometry after gene therapy for DMD	9
CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model	9
Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications	9
Gene therapy corrects the neurological deficits of mice with sialidosis	9
AAV-based gene therapies for neovascular AMD	9
Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice	9
Prime editing: therapeutic advances and mechanistic insights	8
Characterization of anti-AAV2 neutralizing antibody levels in sheep prior to and following intravitreal AAV2.7m8 injection	8
AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis	8
Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential	8
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model	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
AAV8-mediated mouse/human PROC expression rescues thrombophilia in hereditary protein C-deficient mice	7
Acoustically targeted noninvasive gene therapy in large brain volumes	7
Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos	7
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification	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
Genes for bad backs	7
Correction: Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates	7

Correction: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65	7
Codon-optimized human Smad7 gene therapy enhances skeletal muscle mass and function in a murine model of Duchenne muscular dystrophy	7
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement	7