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 2021-04-01 to 2025-04-01.)

Article	Citations
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings	111
Towards ethical drug pricing: the European Orphan Genomic Therapies Fund	100
Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors	59
Distinct functions of CAR-T cells possessing a dectin-1 intracellular signaling domain	53
Automated production of CCR5-negative CD4+-T cells in a GMP-compatible, clinical scale for treatment of HIV-positive patients	49
Micro-dystrophin gene constructs for repairing heart and muscle function in rats: the smaller is enough?	37
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors	36
A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy	35
Multiplex viral tropism assay in complex cell populations with single-cell resolution	34
Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model	30
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	29
CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease	28
Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures	28
Precision ophthalmology: a call for Africa not to be left in the dark	26
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver	26
The impact of heparin and direct thrombin inhibitors on cell-penetrating polymer siRNA transfection	24
Precision medicine: toward restoring fat with gene therapy in inherited lipodystrophy	22
Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells	22
AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial	21
Preclinical evaluation of tissue-selective gene therapies for congenital generalised lipodystrophy	21
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia	20
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice	20
Gene therapy of Dent disease type 1 in newborn ClC-5 null mice for sustained transgene expression and gene therapy effects	20
Correction: Andrew C. G. Porter (1955–2023)	20
Suppression of viral rebound by a Rev-dependent lentiviral particle in SIV-infected rhesus macaques	19

Correction: RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells	18
Gene drives: an alternative approach to malaria control?	17
Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model	17
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury	16
Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation	16
Characterization of integration frequency and insertion sites of adenovirus DNA into mouse liver genomic DNA following intravenous injection	16
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model	15
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation	15
Placental nanoparticle-mediated IGF1 gene therapy corrects fetal growth restriction in a guinea pig model	14
Effect of nanoparticle-mediated delivery of SFRP4 siRNA for treating Dupuytren disease	14
Correction: Antitumoral gene-based strategy involving nitric oxide synthase type III overexpression in hepatocellular carcinoma	14
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage	14
Development of a cGMP-compliant process to manufacture donor-derived, CD45RA-depleted memory CD19-CAR T cells	14
Ghrelin mediated cardioprotection using in vitro models of oxidative stress	13
Schwann cell gene therapies in sight	13
AAV library screening identifies novel vector for efficient transduction of human aorta	13
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model	12
XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma	12
Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo	12
Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease	11
Focused ultrasound widely broadens AAV-delivered Cas9 distribution and activity	11
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute	11
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study	11
AAV vector engineering for human aorta transduction: becoming a smooth operator	11
Spotlight on gene therapy in Germany	10
Control of gene doping in human and horse sports	10
Gene therapy for spinal muscular atrophy: the Qatari experience	10
Stem cell-based therapy for hirschsprung disease, do we have the guts to treat?	10
Long-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-DΔNΔC gene therapy eight-year follow-up of phase I KAT301 study	10
Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing	10
Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells	9
Toward gene therapy of Laron syndrome	9
Upper motor neurons are a target for gene therapy and UCHL1 is necessary and sufficient to improve cellular integrity of diseased upper motor neurons	8
Adeno-associated virus mediated expression of monoclonal antibody MR191 protects mice against Marburg virus and provides long-term expression in sheep	8
Curing SMA: Are we there yet?	8
Safety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases	8
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a	8
Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo	8
Biodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair	8
Intracisternal vs intraventricular injection of AAV1 result in comparable, widespread transduction of the dog brain	8
Rational engineering of a functional CpG-free ITR for AAV gene therapy	8
Induced pluripotent stem cell-derived dendritic cell vaccine therapy genetically modified on the ubiquitin-proteasome system	8
Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice	7
Correction: AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses	7
Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	7
Gene therapy corrects the neurological deficits of mice with sialidosis	7
Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models	7
CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model	7
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system	7
The estimated annual financial impact of gene therapy in the United States	7