Gene Therapy

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 2021-11-01 to 2025-11-01.)
ArticleCitations
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors111
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice62
Correction: PPARγ is essential for protection against nonalcoholic steatohepatitis51
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury47
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia46
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings41
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model39
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease38
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials36
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system33
Codon changes challenge PCR-based gene doping detection33
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy31
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry27
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa23
Implications of maternal-fetal health on perinatal stem cell banking23
Correction: The disparate burden of infectious diseases22
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV222
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production22
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene22
hInGeTox: a human-based in vitro platform to evaluate lentivirus/host interactions that contribute to genotoxicity20
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