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UTX/KDM6A deletion promotes the recovery of spinal cord injury by epigenetically triggering intrinsic neural regeneration

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 20 ()

Interrupted axons that fail to regenerate mainly cause poor recovery after spinal cord injury (SCI). How neurons epigenetically respond to injury dete......

Discovery of key genes as novel biomarkers specifically associated with HPV-negative cervical cancer

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 21 ()

Cervical cancer is a common female malignancy that is mainly caused by human papillomavirus (HPV) infection. However, the incidence of HPV-negative ce......

Ad- and AAV8-mediated ABCA1 gene therapy in a murine model with retinal ischemia/reperfusion injuries

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 20 ()

The anti-inflammatory molecule annexin A1 (ANXA1) determines the ultimate fate of retinal ganglion cell (RGC) in glaucoma. Cytoplasmic and extracellul......

Improved safety of induced pluripotent stem cell-derived antigen-presenting cell-based cancer immunotherapy

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 21 ()

The tumorigenicity and toxicity of induced pluripotent stem cells (iPSCs) and their derivatives are major safety concerns in their clinical applicatio......

The microRNA cluster miR-30b/-30d prevents tumor cell switch from an epithelial to a mesenchymal-like phenotype in GBC

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 20 ()

As a malignancy of the gastrointestinal tract, gallbladder cancer (GBC) continues to exhibit notable rates of mortality. The current study aimed at in......

Serum exosome microRNA panel as a noninvasive biomarker for molecular diagnosis of fulminant myocarditis

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 20 ()

Exosome-derivedmicroRNAs(miRNAs) are potential diagnostic biomarkers. However, little is known about their effectiveness as diagnostic biomarkers of f......

A pro-inflammatory mediator USP11 enhances the stability of p53 and inhibits KLF2 in intracerebral hemorrhage

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 21 ()

Microglial cell activation and neuroinflammation after intracerebral hemorrhage (ICH) lead to secondary brain damage. Ubiquitin-specific protease 11 (......

CRISPR-mediated rapid generation of neural cell-specific knockout mice facilitates research in neurophysiology and pathology

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 20 ()

Inducible conditional knockout mice are important tools for studying gene function and disease therapy, but their generation is costly and time-consum......

Engineering mesenchymal stromal cells with neutralizing and anti-inflammatory capability against SARS-CoV-2 infection

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 21 ()

The emergence of the novel human severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) has led to the pandemic of coronavirus disease 2019 (COV......

Manufacturing NKG2D CAR-T cells with piggyBac transposon vectors and K562 artificial antigen-presenting cells

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 21 ()

Non-viral platforms can be applied rapidly and cost-effectively for chimeric antigen receptor (CAR)-T cell manufacturing. In the present paper, we des......

miR-1254 induced by NESG1 inactivates HDGF/DDX5-stimulated nuclear translocation of beta-catenin and suppresses NPC metastasis

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 20 ()

Nasopharyngeal carcinoma (NPC) is one of the most common malignant tumors in Chinese and other Southeast Asians. We aimed to explore the precise mecha......

Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 20 ()

Adenosine deaminase (ADA) deficiency is an inborn error of metabolism affecting multiple systems and causing severe combined immunodeficiency. We test......

NaCl and KCl mediate log increase in AAV vector particles and infectious titers in a specific/timely manner with the HSV platform

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 21 ()

The increasing demand for adeno-associated virus (AAV) vectors, a result from the surging interest for their potential to cure human genetic diseases ......

Inhibition of indoleamine 2,3-dioxygenase 1 synergizes with oxaliplatin for efficient colorectal cancer therapy

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 20 ()

We investigated the immunogenic cell death provoked by oxaliplatin (OXA) and the involvement of OXA-induced immunosuppression in colorectal cancer. Im......

Genetically blocking HPD via CRISPR-Cas9 protects against lethal liver injury in a pig model of tyrosinemia type I

期刊: MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021; 21 ()

Hereditary tyrosinemia type I (HT1) results from the loss of fumarylacetoacetate hydrolase (FAH) activity and can lead to lethal liver injury (LLI). T......

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