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 2021-08-01 to 2025-08-01.)
ArticleCitations
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma289
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma228
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin210
Promotion or inhibition? This is a question in gene editing205
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference201
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II185
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids182
Recent progress and future challenges in structure-based protein-protein interaction prediction170
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders161
Response to: DNA transposon mechanisms and pathways of genotoxicity155
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification147
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells146
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia145
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques138
From cortex to medulla: Navigating the thymic landscape of T cell education131
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming129
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8124
Reply to In vivo confusion over in vivo conversion124
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer124
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection124
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia122
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors121
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation118
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial115
miRNAs as neuro-oncologic therapeutics: A narrative review112
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery107
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?100
Ad-justing macrophages for cancer immunotherapy100
Targeting EBV gp42 for nasopharyngeal carcinoma prevention99
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations97
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo96
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy96
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors95
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies95
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury94
Generation of highly proliferative, rejuvenated cytotoxic T cell clones through pluripotency reprogramming for adoptive immunotherapy93
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-291
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier90
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans86
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities84
How to democratize cell and gene therapy: A global approach84
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia83
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo80
Biologics-based technologies for highly efficient and targeted RNA delivery80
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities79
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease79
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy77
Late-Breaking Abstracts I74
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act74
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors73
A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency72
Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR2570
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?69
Quo vadis American postdoc?69
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells69
Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling68
Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain68
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing68
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP68
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD868
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy67
Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche67
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain65
Smoking and tetramer tryptase accelerate intervertebral disc degeneration by inducing METTL14-mediated DIXDC1 m6 modification65
Long Noncoding RNA-Maternally Expressed Gene 3 Contributes to Hypoxic Pulmonary Hypertension64
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