OOIR: Observatory of International Research

Papers

(The H4-Index of Molecular Therapy is 63. 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 2021-06-01 to 2025-06-01.)

Article	Citations
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	244
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	220
From cortex to medulla: Navigating the thymic landscape of T cell education	209
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	196
Promotion or inhibition? This is a question in gene editing	189
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	183
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	172
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies	171
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	156
Generation of highly proliferative, rejuvenated cytotoxic T cell clones through pluripotency reprogramming for adoptive immunotherapy	149
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	147
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	143
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	138
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	138
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans	135
miRNAs as neuro-oncologic therapeutics: A narrative review	131
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	130
WDR5 facilitates EMT and metastasis of CCA by increasing HIF-1α accumulation in Myc-dependent and independent pathways	127
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	126
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	120
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	119
Extracellular vesicles in neuroinflammation: Pathogenesis, diagnosis, and therapy	118
Response to: DNA transposon mechanisms and pathways of genotoxicity	118
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	116
Recent progress and future challenges in structure-based protein-protein interaction prediction	115

How to democratize cell and gene therapy: A global approach	115
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	110
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	105
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	102
Biologics-based technologies for highly efficient and targeted RNA delivery	94
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	93
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia	93
Reply to In vivo confusion over in vivo conversion	91
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	91
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	91
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	87
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	86
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	86
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	85
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	85
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	85
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	85
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	84
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	84
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	81
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	80
Late-Breaking Abstracts I	79
In This Issue	79
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	77
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	75
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems	74
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	72
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8	72
Long Noncoding RNA-Maternally Expressed Gene 3 Contributes to Hypoxic Pulmonary Hypertension	71
Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial	71
Retraction Notice to: The YY1-HOTAIR-MMP2 Signaling Axis Controls Trophoblast Invasion at the Maternal-Fetal Interface	69
Targeting PD-1+ T cells with small-format immunocytokines enhances IL-12 antitumor activity	68
Quo vadis American postdoc?	66
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?	66
Low-inflammatory lipid nanoparticles facilitate safe mRNA vaccination against influenza virus infection	65
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells	65
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP	65
Switch of ELF3 and ATF4 transcriptional axis programs the amino acid insufficiency-linked epithelial-to-mesenchymal transition	64
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain	63
Fluoxetine ameliorates mucopolysaccharidosis type IIIA	63
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	63