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 2022-05-01 to 2026-05-01.)

Article	Citations
Ad-justing macrophages for cancer immunotherapy	468
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	322
Response to: DNA transposon mechanisms and pathways of genotoxicity	288
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	281
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	269
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	248
From cortex to medulla: Navigating the thymic landscape of T cell education	188
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	182
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	182
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy	180
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	170
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	164
Precision A3G base editors for disease modeling and correction	156
A synthetic hyperglycemia-sensing gene circuit enhances blood glucose homeostasis in diabetic mice	150
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	148
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	133
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	132
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	127
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	120
ALCAT1 promotes diabetic cardiomyopathy by linking myocardial tetralinoleoyl cardiolipin deficiency to lipotoxicity	119
JMJD3 and UTX as key targets for gene-modified mesenchymal stem cell therapy in cartilage tissue engineering	118
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	113
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	112
CAR-modified marrow infiltrating lymphocytes efficiently target malignant plasma cells with very low antigen density	112
Promotion or inhibition? This is a question in gene editing	111

Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	105
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	105
Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs	102
Biologics-based technologies for highly efficient and targeted RNA delivery	98
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	98
First-in-human intracisternal dosing of RGX-111 in severe MPS I is well-tolerated and generates sustained neurodevelopment without HSCT	97
To make biology programmable, we must master its generative grammar	96
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	90
Taming Autoimmunity: Alpha-1 Antitrypsin Overexpressing Mesenchymal Stem/Stromal Cells Promote Regulatory T Cell Crosstalk to Reverse Diabetes	87
Structural basis of liver de-targeting and neuronal tropism of CNS-targeted AAV capsids	84
CDK4/6 inhibition enhances CAR-T cell therapy in solid tumors	84
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	82
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	81
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	80
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	79
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	78
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	77
Recent progress and future challenges in structure-based protein-protein interaction prediction	77
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	77
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	75
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	75
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	73
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	71
How to democratize cell and gene therapy: A global approach	69
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	69
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	69
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	69
miRNAs as neuro-oncologic therapeutics: A narrative review	67
Molecular Therapy Family Highlights	67
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	67
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes	65
Targeting of p53-Transcriptional Dysfunction by Conditionally Replicating Adenovirus Is Not Limited by p53-Homologues	65
Optogenetic vision restoration in the face of secondary and tertiary remodeling in the rd1 mouse retina	64
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	64
Toward scalable helper T cells from iPSCs	64
Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25	64
Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1	63
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	63