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 2021-08-01 to 2025-08-01.)
ArticleCitations
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors98
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice59
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings58
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury46
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia45
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 disease37
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system35
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy34
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials34
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry33
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV231
Implications of maternal-fetal health on perinatal stem cell banking31
Correction: The disparate burden of infectious diseases27
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa27
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene24
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis23
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production23
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer22
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse21
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice20
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