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 2022-06-01 to 2026-06-01.)
ArticleCitations
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia151
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice72
Correction: PPARĪ³ is essential for protection against nonalcoholic steatohepatitis68
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings65
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors60
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease43
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy35
Beneficial bystander-enhanced cryptic splice rescue of cardiac-type Fabry GLA IVS4+919G>A by adenine base editing in patient fibroblasts33
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system31
Drinkable gene therapy foam for the treatment of constrictive esophageal carcinoma30
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials28
Codon changes challenge PCR-based gene doping detection28
First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study27
The co-delivery of Programmed Death 1 ligands enhances and prolongs rAAV-mediated gene expression in pre-immunized mice26
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV226
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa25
Implications of maternal-fetal health on perinatal stem cell banking24
Correction: The disparate burden of infectious diseases24
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery23
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 immunoprophylaxis22
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production22
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse22
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