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 2022-05-01 to 2026-05-01.)
ArticleCitations
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia146
Correction: PPARĪ³ is essential for protection against nonalcoholic steatohepatitis68
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease67
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings60
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice57
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors51
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy45
Codon changes challenge PCR-based gene doping detection43
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials35
Drinkable gene therapy foam for the treatment of constrictive esophageal carcinoma32
First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study31
Beneficial bystander-enhanced cryptic splice rescue of cardiac-type Fabry GLA IVS4+919G>A by adenine base editing in patient fibroblasts28
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV227
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system27
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery26
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa26
The co-delivery of Programmed Death 1 ligands enhances and prolongs rAAV-mediated gene expression in pre-immunized mice26
Correction: The disparate burden of infectious diseases24
Implications of maternal-fetal health on perinatal stem cell banking24
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production23
Murine toxicology assessment of avgn7.2, a novel gene therapeutic for inclusion body myositis and other muscle wasting diseases22
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis21
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse21
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