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
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia	93
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	93
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	91
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
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	87
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	86
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	86
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	85
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
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
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	63
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain	63
Fluoxetine ameliorates mucopolysaccharidosis type IIIA	63