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 2022-06-01 to 2026-06-01.)

Article	Citations
Ad-justing macrophages for cancer immunotherapy	482
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	328
Response to: DNA transposon mechanisms and pathways of genotoxicity	291
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	253
From cortex to medulla: Navigating the thymic landscape of T cell education	195
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	195
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	184
Precision A3G base editors for disease modeling and correction	183
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	183
A synthetic hyperglycemia-sensing gene circuit enhances blood glucose homeostasis in diabetic mice	167
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	160
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	155
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	151
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	135
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	134
CAR-modified marrow infiltrating lymphocytes efficiently target malignant plasma cells with very low antigen density	130
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	123
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	123
To make biology programmable, we must master its generative grammar	121
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	121
CDK4/6 inhibition enhances CAR-T cell therapy in solid tumors	121
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	115
Structural basis of liver de-targeting and neuronal tropism of CNS-targeted AAV capsids	115
Taming autoimmunity: Alpha-1 antitrypsin overexpressing mesenchymal stromal cells promote regulatory T cell crosstalk to reverse diabetes	113
Promotion or inhibition? This is a question in gene editing	108

Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	107
Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs	104
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	100
Biologics-based technologies for highly efficient and targeted RNA delivery	100
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	98
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	91
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	90
First-in-human intracisternal dosing of RGX-111 in severe MPS I is well tolerated and generates sustained neurodevelopment without HSCT	88
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	85
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	85
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	83
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	82
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	82
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	80
ALCAT1 promotes diabetic cardiomyopathy by linking myocardial tetralinoleoyl cardiolipin deficiency to lipotoxicity	79
miRNAs as neuro-oncologic therapeutics: A narrative review	78
JMJD3 and UTX as key targets for gene-modified mesenchymal stem cell therapy in cartilage tissue engineering	78
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	78
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	76
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy	73
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	73
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	73
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	70
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	70
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	69
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	69
Recent progress and future challenges in structure-based protein-protein interaction prediction	69
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	69
How to democratize cell and gene therapy: A global approach	69
Programmable miRNA-Guided RNA-Toxin Switch for Selective Elimination of Cancer Cells	69
Targeting of p53-Transcriptional Dysfunction by Conditionally Replicating Adenovirus Is Not Limited by p53-Homologues	68
Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25	67
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	66
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	66
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	65
Late-Breaking Abstracts I	64
Disease-modifying, multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques	64
From bench to bloodstream: Lipid nanoparticles drive on-demand CAR-T cells	64
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	64