Gene Therapy

Papers
(The H4-Index of Gene Therapy is 23. 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 2022-08-01 to 2026-08-01.)
ArticleCitations
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice170
Correction: PPARĪ³ is essential for protection against nonalcoholic steatohepatitis76
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors72
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia71
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease62
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings44
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy37
Beneficial bystander-enhanced cryptic splice rescue of cardiac-type Fabry GLA IVS4+919G>A by adenine base editing in patient fibroblasts36
Codon changes challenge PCR-based gene doping detection34
Drinkable gene therapy foam for the treatment of constrictive esophageal carcinoma34
First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study32
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system31
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV230
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production30
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa27
Correction: The disparate burden of infectious diseases26
Murine toxicology assessment of avgn7.2, a novel gene therapeutic for inclusion body myositis and other muscle wasting diseases25
The co-delivery of Programmed Death 1 ligands enhances and prolongs rAAV-mediated gene expression in pre-immunized mice25
Improving the precision of AAV lung gene therapy for SP-B deficiency using computationally derived lung-specific promoters25
Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery24
Implications of maternal-fetal health on perinatal stem cell banking24
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse24
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis23
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