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Versatile and efficient in vivo genome editing with compact Streptococcus pasteurianus Cas9

期刊: MOLECULAR THERAPY, 2022; 30 (1)

Compact CRISPR-Cas9 systems that can be packaged into an adeno-associated virus (AAV) show promise for gene therapy. However, the requirement of proto......

JIF:8.335

Cas12a variants designed for lower genome-wide off-target effect through stringent PAM recognition

期刊: MOLECULAR THERAPY, 2022; 30 (1)

Cas12a is an RNA-guided endonuclease that has been widely used for convenient multiplex gene editing with low off-target effects. To minimize off-targ......

JIF:8.335

circ-EIF6 encodes EIF6-224aa to promote TNBC progression via stabilizing MYH9 and activating the Wnt/beta-catenin pathway

期刊: MOLECULAR THERAPY, 2022; 30 (1)

The protein-coding ability of circular RNAs (circRNAs) has recently been a hot topic, but the expression and roles of protein-coding circRNAs in tripl......

JIF:8.335

Type I IFNs repolarized a CD169+macrophage population with anti-tumor potentials in hepatocellular carcinoma

期刊: MOLECULAR THERAPY, 2022; 30 (2)

Macrophages constitute a major component in human hepatocellular carcinoma (HCC) and perform various functions to facilitate disease progression. Repr......

JIF:8.335

sEVs(RVG) selectively delivers antiviral siRNA to fetus brain, inhibits ZIKV infection and mitigates ZIKV- induced in mouse model

期刊: MOLECULAR THERAPY, 2022; 30 (5)

Zitra virus (ZIKV), a flavivirus associated with neurological disorders, constitutes a global health threat. During pregnancy, ZIKV traverses the plac......

JIF:8.335

Long noncoding RNA LINC01234 promotes hepatocellular carcinoma progression through orchestrating aspartate metabolic reprogramming

期刊: MOLECULAR THERAPY, 2022; 30 (6)

Amino acids metabolism, especially aspartate metabolism, is often altered in human cancers including hepatocellular carcinoma (HCC) and this metabolic......

JIF:8.335

A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors

期刊: MOLECULAR THERAPY, 2022; 30 (8)

The inefficient tumor penetration of therapeutic antibodies has hampered their effective use in treating solid tumors. Here, we report the identificat......

JIF:8.335

KLF16 enhances stress tolerance of colorectal carcinomas by modulating nucleolar homeostasis and translational reprogramming

期刊: MOLECULAR THERAPY, 2022; 30 (8)

Translational reprogramming is part of the unfolded protein response (UPR) during endoplasmic reticulum (ER) stress, which acts to the advantage of ca......

JIF:8.335

ADAR2 increases in exercised heart and protects against myocardial infarction and doxorubicin-induced cardiotoxicity

期刊: MOLECULAR THERAPY, 2022; 30 (1)

Exercise training benefits the heart. The knowledge of posttranscription regulation, especially RNA editing, in hearts remain rare. ADAR2 is an enzyme......

JIF:8.335

Single AAV-mediated CRISPR-Nme2Cas9 efficiently reduces mutant hTTR expression in a transgenic mouse model of transthyretin amyloidosis

期刊: MOLECULAR THERAPY, 2022; 30 (1)

Transthyretin (TTR) amyloidosis is a hereditary life -threatening disease characterized by deposition of amyloid fibrils. The main causes of TTR amylo......

JIF:8.335

Mitochondrial fragmentation is crucial for c-Myc-driven hepatoblastoma-like liver tumors

期刊: MOLECULAR THERAPY, 2022; 30 (4)

Hepatoblastoma is the most common liver cancer in children, and the aggressive subtype often has a poor prognosis and lacks effective targeted therapy......

JIF:8.335

The m6A methyltransferase Mettl3 deficiency attenuates hepatic stellate cell activation and liver fibrosis

期刊: MOLECULAR THERAPY, 2022; 30 (12)

Activation of hepatic stellate cells (HSCs) is a central driver of liver fibrosis. Previous investigations have identified various altered epigenetic ......

JIF:8.335

RNA m(6)A methylation regulates dissemination of cancer cells by modulating expression and membrane localization of beta-catenin

期刊: MOLECULAR THERAPY, 2022; 30 (4)

N6-methyladenosine (m(6)A) methylation, which is modified by the METTL3/METTL14 complex, is a dominant internal modification in mammalian RNA and tigh......

JIF:8.335

CRISPR-mediated MECOM depletion retards tumor growth by reducing cancer stem cell properties in lung squamous cell carcinoma

期刊: MOLECULAR THERAPY, 2022; 30 (11)

Targeted therapy for lung squamous cell carcinoma (LUSC) re-mains a challenge due to the lack of robust targets. Here, we identified MECOM as a candid......

JIF:8.335

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