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 2022-01-01 to 2026-01-01.)

Article	Citations
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	92
Thank you to our 2024 reviewers	79
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	76
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	75
Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	72
Molecular earplugs to protect the inner ear	72
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	62
Genetic surgery for a cystic fibrosis-causing splicing mutation	62
Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles	62
RNA-seq analysis of the human surfactant air-liquid interface culture reveals alveolar type II cell-like transcriptome	62
Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates	51
Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP	50
Delivery of nVEGFi using AAV8 for the treatment of neovascular age-related macular degeneration	49
First use of adeno-associated viruses in the human inner ear	48
Differential T cell immune responses to deamidated adeno-associated virus vector	46
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	46
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	41
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	41
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	40
Exploring human plasma proteomic variations in mucolipidosis type IV	39
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	37
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	36
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	36
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8	36
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies	35

Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa	35
Dose-finding and in vivo safety study of an adipose targeted leptin gene therapy for congenital leptin deficiency	34
AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear	33
Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates	33
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay	33
The unknown impact of conditioning on HSC engraftment and clonal dynamics	31
Modulation of AAV transduction and integration targeting by topoisomerase poisons	31
Optimization of anti-CD19 CAR T cell production for treatment of patients with chronic lymphocytic leukemia	31