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 2021-09-01 to 2025-09-01.)

Article	Citations
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	102
Thank you to our 2024 reviewers	77
Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP	67
Exploring human plasma proteomic variations in mucolipidosis type IV	66
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	65
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	64
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	62
Genetic surgery for a cystic fibrosis-causing splicing mutation	60
Delivery of nVEGFi using AAV8 for the treatment of neovascular age-related macular degeneration	57
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	57
Molecular earplugs to protect the inner ear	55
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	55
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia	53
RNA-seq analysis of the human surfactant air-liquid interface culture reveals alveolar type II cell-like transcriptome	49
First use of adeno-associated viruses in the human inner ear	46
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8	45
Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia	45
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	44
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	41
Safety and efficiency modifications of SIV-based integrase-defective lentiviral vectors for immunization	40
Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	39
Differential T cell immune responses to deamidated adeno-associated virus vector	38
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	38
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	37
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	36

Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa	35
AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear	35
Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice	34
Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy	34
Dose-finding and in vivo safety study of an adipose targeted leptin gene therapy for congenital leptin deficiency	33
Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates	32
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