OOIR: Observatory of International Research

Papers

(The H4-Index of Molecular Therapy is 64. 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
Promotion or inhibition? This is a question in gene editing	347
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	259
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	237
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	229
Reply to In vivo confusion over in vivo conversion	224
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	201
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies	197
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	182
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	178
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	162
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	156
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	152
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans	150
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	147
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	140
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy	138
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	136
Ad-justing macrophages for cancer immunotherapy	134
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	132
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	131
From cortex to medulla: Navigating the thymic landscape of T cell education	128
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	121
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	113
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	110
miRNAs as neuro-oncologic therapeutics: A narrative review	110

Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	109
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	108
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	105
Response to: DNA transposon mechanisms and pathways of genotoxicity	105
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	102
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia	101
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	98
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	96
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	96
Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs	95
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	94
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	90
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	89
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	88
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	87
Biologics-based technologies for highly efficient and targeted RNA delivery	83
How to democratize cell and gene therapy: A global approach	83
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	82
Recent progress and future challenges in structure-based protein-protein interaction prediction	81
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	79
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	78
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	77
Low-inflammatory lipid nanoparticles facilitate safe mRNA vaccination against influenza virus infection	76
Molecular Therapy Family Highlights	76
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	76
Targeting PD-1+ T cells with small-format immunocytokines enhances IL-12 antitumor activity	76
Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1	75
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP	75
Optogenetic vision restoration in the face of secondary and tertiary remodeling in the rd1 mouse retina	72
Molecular Therapy’s growing influence	71
Response: Promise and open questions of optogenetic vision restoration by MCO	70
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	69
Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche	69
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells	68
Quo vadis American postdoc?	67
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?	66
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	65
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing	65
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	64