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 2021-05-01 to 2025-05-01.)

Article	Citations
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	227
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	214
Reply to In vivo confusion over in vivo conversion	210
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	195
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans	185
From cortex to medulla: Navigating the thymic landscape of T cell education	182
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	174
Promotion or inhibition? This is a question in gene editing	168
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	167
Response to: DNA transposon mechanisms and pathways of genotoxicity	152
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia	142
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	140
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	137
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies	136
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	135
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	131
Generation of highly proliferative, rejuvenated cytotoxic T cell clones through pluripotency reprogramming for adoptive immunotherapy	130
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	124
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	122
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	119
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	118
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	116
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	115
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	114
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	113

High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	113
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	113
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	113
miRNAs as neuro-oncologic therapeutics: A narrative review	107
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	104
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	98
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	96
Recent progress and future challenges in structure-based protein-protein interaction prediction	94
How to democratize cell and gene therapy: A global approach	92
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	90
Extracellular vesicles in neuroinflammation: Pathogenesis, diagnosis, and therapy	89
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	85
Biologics-based technologies for highly efficient and targeted RNA delivery	85
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
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	84
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	84
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	83
WDR5 facilitates EMT and metastasis of CCA by increasing HIF-1α accumulation in Myc-dependent and independent pathways	82
In This Issue	82
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems	81
Late-Breaking Abstracts I	81
Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain	80
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	79
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	76
Progress in skin gene therapy: From the inside and out	76
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	76
Managing allorejection in off-the-shelf CAR-engineered cell therapies	74
A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency	74
Targeting cholesterol biosynthesis promotes anti-tumor immunity by inhibiting long noncoding RNA SNHG29-mediated YAP activation	71
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	70
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes	70
Evaluation of repRNA vaccine for induction and in utero transfer of maternal antibodies in a pregnant rabbit model	68
Molecular Therapy Family Highlights	68
Complete remission of tumors in mice with neoantigen-painted exosomes and anti-PD-1 therapy	66
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice	66
ASGCT 27th Annual Meeting Abstracts	65
Response: Promise and open questions of optogenetic vision restoration by MCO	64
Autologous transplantation of mitochondria/rAAV IGF-I platforms in human osteoarthritic articular chondrocytes to treat osteoarthritis	64