Objective:This study aims to analyze the characteristics and current status of gene therapy drug policy documents and to provide recommendations for optimizing China's gene therapy drug policy system.Methods:A three-dimensional analytical framework of"policy instruments-interactive subjects-policy phases"was constructed using content analysis and word frequency analysis.This framework was applied to the relevant policy documents issued from March 2009 to March 2024,enabling multidimensional classification and cross-comparison.Results:Among the 37 national-level policy documents included,246 text items were identified under the dimension of policy instruments,with supply-based instruments(45.9%)and environmental instruments(41.5%)being more prevalent,while demand-based instruments(12.6%)were less represented.The distribution of policy instruments was skewed,and internal structural differences were observed.In the dimension of interactive subjects,195 text items were identified,with drug manufacturing and R&D enterprises(36.9%),drug regulatory and accreditation agencies(22.1%),patients and subjects(19.0%),medical institutions and healthcare professionals(11.8%),and other government departments(10.2%)playing uneven roles.In the dimension of policy phases,the policies were categorized into three stages:drug R&D,production,and usage,with some policies covering multiple phases.Conclusions and suggestions:The study suggests combining and optimizing policy instruments for balanced application,strengthening demand-oriented policies,considering the needs of different interacting entities to formulate comprehensive policies,promoting cross-subject collaboration for win-win outcomes,and enhancing phase-linkage efficiency to build a comprehensive chain ecosystem for gene therapy drug development.

OBJECTIVETo determine the expression level of Smad ubiquitination regulatory factor 1 (SMURF1) gene in hepatocellular carcinoma, and to explore its role in liver cancer.

METHODSWith non-neoplastic adjacent normal tissues as controls, real-time PCR and Western blotting were used for measuring the expression of SMURF1 mRNA and protein in 89 samples of hepatocellular carcinoma. Correlations between SMURF1 expression and clinical features were explored. Following transfection of SMURF1--specific small interference RNA(siRNA), the apoptosis and proliferation of hepatic cancer cells Hep G2 were detected using flow cytometry and MTT assays .

RESULTSThe expression of SMURF1 mRNA and protein were significantly higher in hepatocellular cancer tissues compared with the paired normal tissues (P< 0.05). The expression of SMURF1 however did not correlate with any clinical features (P> 0.05). Transfection of SMURF1-specific siRNA can promote the apoptosis whilst inhibit the proliferation of Hep G2 cells.

CONCLUSIONThe expression of SMURF1 is enhanced in hepatocellular carcinoma, which may have played a role in the disease through affecting apoptosis and proliferation of hepatic cancer cells.

Female ; Hep G2 Cells ; Humans ; Liver Neoplasms ; pathology ; Male ; RNA, Messenger ; analysis ; Transforming Growth Factor beta ; physiology ; Ubiquitin-Protein Ligases ; genetics ; physiology

Aged ; Atrial Fibrillation ; surgery ; Catheter Ablation ; adverse effects ; Humans ; Male ; Pulmonary Veno-Occlusive Disease ; etiology ; therapy

Objective To study the situation of 1-5 years old children's antibody against Coxsackieviras A group 16 strain( CVA16 )in Guangdong, Heilongjiang, Yunnan Province and Xinjiang Uygur Autonomous Regions, China,2005, it can offer scientific evidences for preventing and controlling CVA16 causative hand-food and mouth disease. Methods Using microneutrilisation test, to study 503 serum samples randomly selected from sera collected in 2005.Resulte. Positive rate of anti-CVA16 antibody were 41.90%, 9.40%, 40.00% and 34.40% in Gnangdong, Heilongjiang, Yunnan and Xinjiang, respectively. Antibody titer was relative low( average, 1:6.1 ) and there was no statistical difference of geometry mean of antibody titer (GMT) among Guangdong, Heilongjiang, Yunnan ( F = 0.97,0.40, 1.06, respectively; P > 0.05 ), while there had statistical difference of GMT between Heilongjiang and other three regions ( F = 10.61, P < 0.00). Conclusions There had probably existed local epidemic in some regions of Gnangdong, Heilongjiang, Yunnan Province and Xinjiang Uygur Autonomous Regions, China, 2005 or even before, but the area and degree of transmission and epidemic had difference. Children aged from 1-5 years old were relatively susceptible population of CVA16 infection.

Heat stroke(HS)is a serious life-threatening disease caused by heat injury and characterized by a core body temperature>40℃with central nervous system dysfunction and multi-organ failure.The main pathophysiological manifestations of HS are the thermal acute phase response and thermoregulatory imbalance.Proteins are particularly sensitive to heat,and the thermal environment can cause massive protein denaturation,resulting in the deposition of unfolded and misfolded proteins in the cytoplasm,causing cellular dysfunction and even death.The unfolded protein response(UPR),mainly divided into the endoplasmic reticulum UPR and the mitochondrial UPR,is an important physiological process that helps proteins to fold correctly or degrade irretrievably denatured proteins.This paper summarizes the regulatory mechanisms of UPR,the relationship between UPR and severe diseases,as well as the relationship between HS and UPR to provide new ideas for the treatment of HS.