Molecular Therapy

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 2022-01-01 to 2026-01-01.)
ArticleCitations
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference385
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation280
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial252
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans249
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids243
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities200
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders186
Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs170
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II160
Response to: DNA transposon mechanisms and pathways of genotoxicity159
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo152
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells150
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies143
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery143
From cortex to medulla: Navigating the thymic landscape of T cell education136
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations136
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma135
Reply to In vivo confusion over in vivo conversion135
Biologics-based technologies for highly efficient and targeted RNA delivery133
Targeting EBV gp42 for nasopharyngeal carcinoma prevention125
Ad-justing macrophages for cancer immunotherapy123
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?119
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques118
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier112
How to democratize cell and gene therapy: A global approach111
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin111
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy107
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo105
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors103
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia103
Promotion or inhibition? This is a question in gene editing100
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer100
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy99
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification93
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy92
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia92
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection90
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-288
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB886
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors86
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia86
miRNAs as neuro-oncologic therapeutics: A narrative review85
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma84
Recent progress and future challenges in structure-based protein-protein interaction prediction84
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease84
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury84
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities82
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming77
Low-inflammatory lipid nanoparticles facilitate safe mRNA vaccination against influenza virus infection76
Molecular Therapy Family Highlights75
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells74
Retraction Notice to: The YY1-HOTAIR-MMP2 Signaling Axis Controls Trophoblast Invasion at the Maternal-Fetal Interface73
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?73
Quo vadis American postdoc?73
Response: Promise and open questions of optogenetic vision restoration by MCO71
From bench to bloodstream: Lipid nanoparticles drive on-demand CAR-T cells71
ANKRD22 promotes resolution of psoriasiform skin inflammation by antagonizing NIK-mediated IL-23 production69
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells69
Autologous transplantation of mitochondria/rAAV IGF-I platforms in human osteoarthritic articular chondrocytes to treat osteoarthritis68
Progress in skin gene therapy: From the inside and out68
Late-Breaking Abstracts I67
Long Noncoding RNA-Maternally Expressed Gene 3 Contributes to Hypoxic Pulmonary Hypertension65
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing64
Molecular Therapy’s growing influence64
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