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 2022-05-01 to 2026-05-01.)

Article	Citations
Thank you to our 2024 reviewers	108
Exploring human plasma proteomic variations in mucolipidosis type IV	99
Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	96
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	92
Molecular earplugs to protect the inner ear	90
First use of adeno-associated viruses in the human inner ear	90
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	85
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	73
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8	72
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	69
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	67
Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP	54
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	48
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	45
Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles	42
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	42
Genetic surgery for a cystic fibrosis-causing splicing mutation	41
Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates	41
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	41
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	40
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	40
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay	40
Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells	39
Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa	38
An investigation of the immune epitopes of adeno-associated virus capsid-derived peptides among hemophilia patients	38

Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy	37
Molecular Therapy Advances: Building the bridge between discovery and cure	36
The unknown impact of conditioning on HSC engraftment and clonal dynamics	36
Modulation of AAV transduction and integration targeting by topoisomerase poisons	34
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development	33
Efficacy of HSV-TK/GCV system suicide gene therapy using SHED expressing modified HSV-TK against lung cancer brain metastases	32
Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates	32
AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear	32