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

Article	Citations
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	306
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	238
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	219
Promotion or inhibition? This is a question in gene editing	215
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	206
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	192
From cortex to medulla: Navigating the thymic landscape of T cell education	176
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	170
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	161
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	149
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia	148
Reply to In vivo confusion over in vivo conversion	147
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	141
Response to: DNA transposon mechanisms and pathways of genotoxicity	138
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	130
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	129
Biologics-based technologies for highly efficient and targeted RNA delivery	129
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	128
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	126
How to democratize cell and gene therapy: A global approach	126
Recent progress and future challenges in structure-based protein-protein interaction prediction	124
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	121
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	120
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	108
Ad-justing macrophages for cancer immunotherapy	102

The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	101
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	101
Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs	100
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies	100
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	99
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	99
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	97
Generation of highly proliferative, rejuvenated cytotoxic T cell clones through pluripotency reprogramming for adoptive immunotherapy	97
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	92
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	91
miRNAs as neuro-oncologic therapeutics: A narrative review	91
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	87
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	86
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	85
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	85
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	85
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans	84
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy	84
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	84
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	83
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	78
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	76
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	76
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	74
Late-Breaking Abstracts I	73
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	72
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	71
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8	70
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing	70
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?	69
Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain	69
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells	68
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	68
Quo vadis American postdoc?	68
Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche	67
Complete remission of tumors in mice with neoantigen-painted exosomes and anti-PD-1 therapy	65
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP	65
Molecular Therapy’s growing influence	64
Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling	64
Intra-arterial transplantation of autologous mesoangioblasts in m.3243A>G mutation carriers is safe: First phase 1/2 human clinical study	64