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 2022-08-01 to 2026-08-01.)

Article	Citations
Response to: DNA transposon mechanisms and pathways of genotoxicity	522
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	349
From cortex to medulla: Navigating the thymic landscape of T cell education	304
Precision A3G base editors for disease modeling and correction	276
A synthetic hyperglycemia-sensing gene circuit enhances blood glucose homeostasis in diabetic mice	219
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	209
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	208
To make biology programmable, we must master its generative grammar	199
Promotion or inhibition? This is a question in gene editing	190
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	180
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	175
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	160
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	155
miRNAs as neuro-oncologic therapeutics: A narrative review	142
CAR-modified marrow infiltrating lymphocytes efficiently target malignant plasma cells with very low antigen density	139
First-in-human intracisternal dosing of RGX-111 in severe MPS I is well tolerated and generates sustained neurodevelopment without HSCT	138
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	136
Taming autoimmunity: Alpha-1 antitrypsin overexpressing mesenchymal stromal cells promote regulatory T cell crosstalk to reverse diabetes	133
Structural basis of liver de-targeting and neuronal tropism of CNS-targeted AAV capsids	132
CDK4/6 inhibition enhances CAR-T cell therapy in solid tumors	132
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	121
Ad-justing macrophages for cancer immunotherapy	119
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	117
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	108
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	104

Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs	101
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	101
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	98
Targeting of Aberrant αvβ6 Integrin Expression in Solid Tumors Using Chimeric Antigen Receptor-Engineered T Cells	96
Programmable miRNA-guided RNA-toxin switch for selective elimination of cancer cells	95
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	94
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	92
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	92
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	84
C9orf72-associated poly-GR in skeletal muscle leads to neuromuscular junction deficits and muscle atrophy	83
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	82
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	81
How to democratize cell and gene therapy: A global approach	80
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	80
ALCAT1 promotes diabetic cardiomyopathy by linking myocardial tetralinoleoyl cardiolipin deficiency to lipotoxicity	80
JMJD3 and UTX as key targets for gene-modified mesenchymal stem cell therapy in cartilage tissue engineering	79
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	78
Biologics-based technologies for highly efficient and targeted RNA delivery	76
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	75
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	75
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	74
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	73
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy	73
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	72
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	72
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	72
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	72
Recent progress and future challenges in structure-based protein-protein interaction prediction	71
Plugging the diastolic calcium leak: MYBPC3 fragment for CPVT gene therapy	71
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	70
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	68
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	67
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	66
Targeting of p53-Transcriptional Dysfunction by Conditionally Replicating Adenovirus Is Not Limited by p53-Homologues	66
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	65
Inflammatory mediators of mRNA vaccine-induced adverse reactions in mice	64
Late-Breaking Abstracts I	64
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems	64
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	64
A safer path to gene correction in junctional epidermolysis bullosa	64