A5101662788- CRISPR and Genetic Engineering
- Virus-based gene therapy research
- RNA Interference and Gene Delivery
- Lung Cancer Treatments and Mutations
- Immunotherapy and Immune Responses
- Advanced biosensing and bioanalysis techniques
- Retinal Development and Disorders
- RNA regulation and disease
- CAR-T cell therapy research
- Cancer Immunotherapy and Biomarkers
- Peptidase Inhibition and Analysis
- Pluripotent Stem Cells Research
- Cancer, Hypoxia, and Metabolism
- Galectins and Cancer Biology
- Viral Infections and Immunology Research
- Glycosylation and Glycoproteins Research
- Immune Cell Function and Interaction
- Cancer Research and Treatments
- Circular RNAs in diseases
- Ubiquitin and proteasome pathways
- Angiogenesis and VEGF in Cancer
- Animal Genetics and Reproduction
- Sexual function and dysfunction studies
- RNA Research and Splicing
- MicroRNA in disease regulation
Wenzhou Medical University
2023-2025
Harbin Medical University
2024-2025
Minzu University of China
2025
State Ethnic Affairs Commission
2025
Beijing University of Chinese Medicine
2025
Sichuan University
2015-2024
State Key Laboratory of Biotherapy
2014-2024
West China Hospital of Sichuan University
2014-2024
West China Second University Hospital of Sichuan University
2023-2024
State Key Laboratory of Oral Diseases
2012-2024
The prime editor (PE) can edit genomes with almost any intended changes, including all 12 possible types of base substitutions, small insertions and deletions, their combinations, without the requirement for double strand breaks or exogenous donor templates. PE demonstrates possibility correcting a variety disease-causing mutations might expand therapeutic application gene editing. In this study, was optimized based on dual-adeno-associated virus (AAV) split-intein system in vitro by...
Abstract In β-thalassemia, either γ-globin induction to form fetal hemoglobin (α2γ2) or β-globin repair restore adult (α2β2) could be therapeutic. ABE8e, a recently evolved adenine base editor variant, can achieve efficient conversion, yet its application in patient-derived hematopoietic stem cells needs further exploration. Here, we purified ABE8e for ribonucleoprotein electroporation of β-thalassemia patient CD34 + and progenitor introduce nucleotide substitutions that upregulate...
Failure of clinical trials nonviral vector-mediated gene therapy arises primarily from either an insufficient transgene expression level or immunostimulation concerns caused by the genetic information carrier (e.g., bacteria-generated, double-stranded DNA (dsDNA)). Neither these issues could be addressed through engineering-sophisticated delivery vehicles. Therefore, we propose a systemic chemically modified messenger RNA (mRNA) as alternative to plasmid (pDNA) in cancer therapy. Modified...
Targeted delivery remains the major challenge for application of small interfering RNA (siRNA). We have developed a lipid/calcium/phosphate (LCP) nanoparticle (NP) to improve siRNA efficiency. The LCP NP was prepared by using microemulsion technology form calcium/phosphate (CaP) core and further coated with cationic lipids. final grafted polyethylene glycol (PEG) anisamide (AA) ligand on surface target sigma receptor-expressing B16F10 melanoma cells. exhibited 40 nm particle size, +25 mV...
Immunotherapy based on the immune checkpoint blockade has emerged as most promising approach for cancer therapy. However, proportion of colorectal patients who benefit from immunotherapy is small due to immunosuppressive tumor microenvironment. Hence, combination an ideal strategy overcome this limitation. In study, we developed a novel CSF-1R (colony-stimulating factor 1 receptor) inhibitor (PLX3397), oncolytic viruses, and anti-PD-1 antibody. Our results demonstrated that triple treatment...
SARI, also called as BATF2, belongs to the BATF family and has been implicated in cancer cell growth inhibition. However, role mechanism of SARI tumour angiogenesis are elusive. Here we demonstrate that deficiency facilitates AOM/DSS-induced colonic tumorigenesis mice. We show is a novel inhibitor colon Antibody array HUVEC-related assays indicate VEGF an essential SARI-controlled inhibition angiogenesis. Furthermore, Co-IP/PAGE/mass spectrometry indicates directly targets ceruloplasmin...
Retinitis pigmentosa (RP) is a group of retinal diseases that cause the progressive death photoreceptor cells and eventually blindness. Mutations in β-domain phosphodiesterase 6 (Pde6b) gene are most identified causes autosomal recessive RP. Clinically, there no effective treatment so far can stop progression RP restore vision. Here, we report base editing approach which adeno-associated virus (AAV)-mediated adenine editor (ABE) delivering to postmitotic photoreceptors was conducted correct...
VEGF receptor 2 (VEGFR2) inhibitors, as efficient antiangiogenesis agents, have been applied in the cancer treatment. However, currently most of these anticancer drugs suffer some adverse effects. Discovery novel VEGFR2 inhibitors drug candidates is still needed.In this investigation, we adopted a restricted de novo design method to inhibitors. We selected potent compound SKLB1002 and analyzed its inhibitory effects on human umbilical vein endothelial cells (HUVEC) vitro. Tumor xenografts...
Non-small cell lung cancer (NSCLC) is the leading cause of cancer-related death. To explore potential small interfering RNA (siRNA) therapy for NSCLC, we have developed anisamide-targeted LCP to efficiently deliver siRNA into cytoplasm sigma receptor-expressing NSCLC cells. Targeted demonstrated a 9-fold higher delivery efficiency compared nontargeted in A549 cells vitro. simultaneously target multiple oncogenic mechanisms, coformulated three sequences targeting HDM2, c-myc and VEGF...
Sufficient mucosal permeability is the bottleneck problem in developing an efficient intestinal delivery system of insulin. Cell-penetrating peptide-based nanocomplexes for enhanced permeation insulin were developed this study. Penetratin, a cell-penetrating peptide was site-specifically modified with bis-β-cyclodextrin group. Insulin-loaded prepared by self-assembly using penetratin or its derivative (P-bis-CD). A stronger intermolecular interaction and higher complex stability observed...
AAV- and CRISPR-Cas9–mediated gene targeting in neonatal mice led to clinical benefits a mouse model of OTC deficiency.
Centrosomal protein dysfunction might cause ciliopathies. However, the role of centrosomal proteins in male infertility remains poorly defined. Here, we identified a pathogenic splicing mutation CEP78 infertile patients with severely reduced sperm number and motility, typical multiple morphological abnormalities flagella phenotype. We further created Cep78 knockout mice, which showed an extremely low count, completely aberrant morphology, approximately null motility. The mice could not be...