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 2022-06-01 to 2026-06-01.)

Article	Citations
Thank you to our 2024 reviewers	118
Exploring human plasma proteomic variations in mucolipidosis type IV	109
Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	100
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	94
Molecular earplugs to protect the inner ear	92
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	90
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8	86
Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates	76
Genetic surgery for a cystic fibrosis-causing splicing mutation	74
Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles	73
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	69
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	56
Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP	56
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	49
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	46
First use of adeno-associated viruses in the human inner ear	43
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	43
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	42
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	42
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	41
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	41
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay	41
Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells	40
An investigation of the immune epitopes of adeno-associated virus capsid-derived peptides among hemophilia patients	40
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy	39

Molecular Therapy Advances: Building the bridge between discovery and cure	38
The unknown impact of conditioning on HSC engraftment and clonal dynamics	37
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies	37
Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice	36
Modulation of AAV transduction and integration targeting by topoisomerase poisons	35
Efficacy and muscle safety assessment of fukutin-related protein gene therapy	33
Dose-finding and in vivo safety study of an adipose targeted leptin gene therapy for congenital leptin deficiency	33
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development	33