OOIR: Observatory of International Research

Papers

(The H4-Index of Molecular Therapy-Methods & Clinical Development is 32. 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 2021-11-01 to 2025-11-01.)

Article	Citations
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	105
Thank you to our 2024 reviewers	89
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	74
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	71
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	71
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	69
Delivery of nVEGFi using AAV8 for the treatment of neovascular age-related macular degeneration	67
RNA-seq analysis of the human surfactant air-liquid interface culture reveals alveolar type II cell-like transcriptome	67
First use of adeno-associated viruses in the human inner ear	63
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	59
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia	57
Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP	57
Molecular earplugs to protect the inner ear	55
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	48
Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	47
Safety and efficiency modifications of SIV-based integrase-defective lentiviral vectors for immunization	47
Genetic surgery for a cystic fibrosis-causing splicing mutation	45
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8	45
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	41
Exploring human plasma proteomic variations in mucolipidosis type IV	40
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	40
Differential T cell immune responses to deamidated adeno-associated virus vector	39
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	38
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	37
Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy	36

Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa	36
An investigation of the immune epitopes of adeno-associated virus capsid-derived peptides among hemophilia patients	35
Efficacy and muscle safety assessment of fukutin-related protein gene therapy	34
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development	33
The unknown impact of conditioning on HSC engraftment and clonal dynamics	33
Efficacy of HSV-TK/GCV system suicide gene therapy using SHED expressing modified HSV-TK against lung cancer brain metastases	32
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies	32
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy	32