OOIR: Observatory of International Research

Papers

(The H4-Index of Gene Therapy is 20. 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 2021-08-01 to 2025-08-01.)

Article	Citations
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors	98
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	58
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury	46
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia	45
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model	39
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	37
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system	35
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials	34
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy	34
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry	33
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2	31
Implications of maternal-fetal health on perinatal stem cell banking	31
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa	27
Correction: The disparate burden of infectious diseases	27
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene	24
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production	23
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis	23
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer	22
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	21
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice	20