Gene Therapy

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 2021-05-01 to 2025-05-01.)
ArticleCitations
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model102
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors68
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice54
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury50
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings39
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia39
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
Gene therapy for spinal muscular atrophy: the Qatari experience33
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials33
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry31
Control of gene doping in human and horse sports30
Correction: The disparate burden of infectious diseases28
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene28
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV226
Viral vector gene delivery of the novel chaperone protein SRCP1 to modify insoluble protein in in vitro and in vivo models of ALS25
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa24
Implications of maternal-fetal health on perinatal stem cell banking24
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis22
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse22
Unlocking patient access to gene therapy: five key practices22
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer22
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