OOIR: Observatory of International Research

Papers

(The H4-Index of Molecular Therapy-Methods & Clinical Development is 33. 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 2021-05-01 to 2025-05-01.)

Article	Citations
Molecular earplugs to protect the inner ear	89
RNA-seq analysis of the human surfactant air-liquid interface culture reveals alveolar type II cell-like transcriptome	85
Safety and efficiency modifications of SIV-based integrase-defective lentiviral vectors for immunization	72
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	64
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia	61
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	56
Thank you to our 2024 reviewers	56
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	55
Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP	52
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	52
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	50
Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	49
First use of adeno-associated viruses in the human inner ear	49
Delivery of nVEGFi using AAV8 for the treatment of neovascular age-related macular degeneration	48
Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system	47
Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia	46
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	45
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	45
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	44
Exploring Human Plasma Proteomic Variations in Mucolipidosis Type IV	43
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	42
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8	42
Differential T cell immune responses to deamidated adeno-associated virus vector	40
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	40
Genetic surgery for a cystic fibrosis-causing splicing mutation	39

Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy	38
Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids	38
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	37
The unknown impact of conditioning on HSC engraftment and clonal dynamics	35
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay	35
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies	35
Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells	35
Improvement of HSV-1 based amplicon vectors for a safe and long-lasting gene therapy in non-replicating cells	34
Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa	33
Modulation of AAV transduction and integration targeting by topoisomerase poisons	33
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development	33