Molecular Therapy is the leading international journal for research on the development of molecular and cellular therapeutics to correct genetic and acquired diseases, including but not limited to research on gene transfer and editing, vector development and design, stem cell manipulation, vaccine development, pre-clinical target validation, safety/efficacy studies, and clinical trials.
《分子治疗》是国际领先的分子和细胞治疗学研究期刊，旨在纠正遗传性和获得性疾病，包括但不限于基因转移和编辑、载体开发和设计、干细胞操作、疫苗开发、临床前靶点验证、安全性/有效性研究和临床试验。
Promoter Methylation-Regulated miR-145-5p Inhibits Laryngeal Squamous Cell Carcinoma Progression by Targeting FSCN1
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.09.018
Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.03.007
Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.12.005
A NeuroD1 AAV-Based Gene Therapy for Functional Brain Repair after Ischemic Injury through In Vivo Astrocyte-to-Neuron Conversion
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.09.003
In Vivo PET Tracking of 89Zr-Labeled Vγ9Vδ2 T Cells to Mouse Xenograft Breast Tumors Activated with Liposomal Alendronate
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.10.006
ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.10.018
Development of Novel Micro-dystrophins with Enhanced Functionality
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.01.002
In Vivo Delivery of a DNA-Encoded Monoclonal Antibody Protects Non-human Primates against Zika Virus
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.03.005
mRNA-Based Protein Replacement Therapy for the Heart
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.11.018
Therapeutic Potential of Wnt and Notch Signaling and Epigenetic Regulation in Mammalian Sensory Hair Cell Regeneration
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.03.017
Long Non-coding RNA ECRAR Triggers Post-natal Myocardial Regeneration by Activating ERK1/2 Signaling
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.10.021
miR-183-5p Inhibits Occurrence and Progression of Acute Myeloid Leukemia via Targeting Erbin
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.01.016
Preclinical Evaluation of ADVM-022, a Novel Gene Therapy Approach to Treating Wet Age-Related Macular Degeneration
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.11.003
MicroRNA-621 Acts as a Tumor Radiosensitizer by Directly Targeting SETDB1 in Hepatocellular Carcinoma
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.11.005
Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.03.001
A Neutralizing Aptamer to TGFBR2 and miR-145 Antagonism Rescue Cigarette Smoke- and TGF-β-Mediated CFTR Expression
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.11.017
Anti-drug Antibody Responses Impair Prophylaxis Mediated by AAV-Delivered HIV-1 Broadly Neutralizing Antibodies
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.01.004
miR-577 Regulates TGF-β Induced Cancer Progression through a SDPR-Modulated Positive-Feedback Loop with ERK-NF-κB in Gastric Cancer
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.02.002
Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice
来源期刊：Molecular TherapyDOI：10.1016/J.YMTHE.2019.05.021
Development of Novel DNA-Encoded PCSK9 Monoclonal Antibodies as Lipid-Lowering Therapeutics
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.10.016
Therapeutic Delivery of miR-148a Suppresses Ventricular Dilation in Heart Failure
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2018.11.011
ICOS Costimulation at the Tumor Site in Combination with CTLA-4 Blockade Therapy Elicits Strong Tumor Immunity
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.07.013
Enhanced Potency of GalNAc-Conjugated Antisense Oligonucleotides in Hepatocellular Cancer Models
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.06.009
Systemic Blockade of ACVR2B Ligands Protects Myocardium from Acute Ischemia-Reperfusion Injury
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.01.013
Synthetic miR-143 Exhibited an Anti-Cancer Effect via the Downregulation of K-RAS Networks of Renal Cell Cancer Cells In Vitro and In Vivo
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.03.004
Oral Gavage of Ginger Nanoparticle-Derived Lipid Vectors Carrying Dmt1 siRNA Blunts Iron Loading in Murine Hereditary Hemochromatosis
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.01.003
CRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editors
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.05.013
lncRNA RMST Enhances DNMT3 Expression through Interaction with HuR
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.09.024
The SINE Compound KPT-350 Blocks Dystrophic Pathologies in DMD Zebrafish and Mice
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.08.016
Weak Agonistic LPS Restores Intestinal Immune Homeostasis
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.07.007
CD8+CD103+ iTregs Inhibit Chronic Graft-versus-Host Disease with Lupus Nephritis by the Increased Expression of CD39
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.07.014
Potentiating Oncolytic Virus-Induced Immune-Mediated Tumor Cell Killing Using Histone Deacetylase Inhibition
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.04.008
Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.06.015
Engineering of a Lectibody Targeting High-Mannose-Type Glycans of the HIV Envelope
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.07.021
Targeted Activation of Cystic Fibrosis Transmembrane Conductance Regulator
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.07.002
Steric Inhibition of 5′ UTR Regulatory Elements Results in Upregulation of Human CFTR
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.06.016
UTX/KDM6A Deletion Promotes Recovery of Spinal Cord Injury by Epigenetically Regulating Vascular Regeneration
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.08.009
First-in-Human Intravenous Seprehvir in Young Cancer Patients: A Phase 1 Clinical Trial
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.08.020
Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.04.014
Endogenously Expressed Antigens Bind Mammalian RNA via Cationic Domains that Enhance Priming of Effector CD8 T Cells by DNA Vaccination
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.01.011
The Role of MicroRNA-21 in Venous Neointimal Hyperplasia: Implications for Targeting miR-21 for VNH Treatment
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.06.011
Anti-inflammatory Activity of MTL-CEBPA, a Small Activating RNA Drug, in LPS-Stimulated Monocytes and Humanized Mice
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.02.018
Malignant Glioma Therapy by Vaccination with Irradiated C6 Cell-Derived Microvesicles Promotes an Antitumoral Immune Response
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.05.016
Rescue from Stx2-Producing E. coli-Associated Encephalopathy by Intravenous Injection of Muse Cells in NOD-SCID Mice
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.09.023
Repurposing an HIV Drug for Zika Virus Therapy
来源期刊：Molecular TherapyDOI：10.1016/j.ymthe.2019.10.004
Addgene: Molecular Therapy Interview with Melina Fan and Karen Guerin
来源期刊：Molecular TherapyDOI：10.1016/J.YMTHE.2018.12.001
