OOIR: Observatory of International Research

Papers

(The H4-Index of Gene Therapy is 22. 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 2021-06-01 to 2025-06-01.)

Article	Citations
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model	78
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors	57
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice	53
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury	41
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia	39
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings	37
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	35
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system	35
Gene therapy for spinal muscular atrophy: the Qatari experience	33
Control of gene doping in human and horse sports	32
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials	31
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry	30
Implications of maternal-fetal health on perinatal stem cell banking	29
Correction: The disparate burden of infectious diseases	27
Viral vector gene delivery of the novel chaperone protein SRCP1 to modify insoluble protein in in vitro and in vivo models of ALS	26
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa	24
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene	23
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2	23
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis	23
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1	22
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	22
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer	22