OOIR: Observatory of International Research

Papers

(The H4-Index of Gene Therapy is 21. 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
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials	34
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry	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
hInGeTox: a human-based in vitro platform to evaluate lentivirus/host interactions that contribute to genotoxicity	21
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