Gene Therapy

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 2022-01-01 to 2026-01-01.)
ArticleCitations
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice123
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model62
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors55
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury53
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia49
Correction: PPARγ is essential for protection against nonalcoholic steatohepatitis44
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings44
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease40
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy39
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system37
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry34
Codon changes challenge PCR-based gene doping detection34
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials30
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa27
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production24
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene23
Murine toxicology assessment of avgn7.2, a novel gene therapeutic for inclusion body myositis and other muscle wasting diseases23
Correction: The disparate burden of infectious diseases23
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery23
Implications of maternal-fetal health on perinatal stem cell banking21
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV221
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse21
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