OOIR: Observatory of International Research

Papers

(The H4-Index of Molecular Therapy-Methods & Clinical Development is 31. 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 2021-04-01 to 2025-04-01.)

Article	Citations
Molecular earplugs to protect the inner ear	88
Detection and quantification of integrated vector copy number by multiplex droplet digital PCR in dual-transduced CAR T cells	83
Oxidative stress induced by sustained supraphysiological intrastriatal GDNF delivery is prevented by dose regulation	67
Generation of hepatitis C virus–resistant liver cells by genome editing–mediated stable expression of RNA aptamer	59
Breaching the blood-brain barrier: AAV triggers dose-dependent toxicity in the brain	59
Evaluation of parameters for efficient purification and long-term storage of herpes simplex virus-based vectors	56
Engineering new metabolic pathways in isolated cells for the degradation of guanidinoacetic acid and simultaneous production of creatine	53
AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency	53
Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease	51
Neurofilament light chain and dorsal root ganglia injury after adeno-associated virus 9 gene therapy in nonhuman primates	49
Large-scale manufacturing of base-edited chimeric antigen receptor T cells	47
Insights in AAV-mediated antigen-specific immunity and a strategy for AAV vaccine dose reduction through AAV-extracellular vesicle association	46
Highly branched poly β-amino ester/CpG-depleted CFTR plasmid nanoparticles for non-viral gene therapy in lung cystic fibrosis disease	46
Characterization of drusen formation in a primary porcine tissue culture model of dry AMD	46
Thank you to our 2024 reviewers	46
Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease	43
αFAP-specific nanobodies mediate a highly precise retargeting of modified AAV2 capsids thereby enabling specific transduction of tumor tissues	41
Cryopreserved anti-CD22 and bispecific anti-CD19/22 CAR T cells are as effective as freshly infused cells	41
Packaging cells for lentiviral vectors generated using the cumate and coumermycin gene induction systems and nanowell single-cell cloning	41
A third dose of the unmodified COVID-19 mRNA vaccine CVnCoV enhances quality and quantity of immune responses	40
DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34+ cell clonogenic potential	39
Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette	39
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia	38
Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series	38
miRNA-mediated control of exogenous OCT4 during mesenchymal-epithelial transition increases measles vector reprogramming efficiency	37

Gene therapy for Friedreich ataxia: Too much, too little, or just right?	37
AUF1 gene transfer increases exercise performance and improves skeletal muscle deficit in adult mice	36
Safety and efficiency modifications of SIV-based integrase-defective lentiviral vectors for immunization	35
SAG therapy restores bone growth and reduces enchondroma incidence in a model of skeletal chondrodysplasias caused by Ihh deficiency	35
RNA-seq analysis of the human surfactant air-liquid interface culture reveals alveolar type II cell-like transcriptome	33
Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model	33
Follistatin-like 1 promotes proliferation of matured human hypoxic iPSC-cardiomyocytes and is secreted by cardiac fibroblasts	31