1.Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile metachromatic leukodystrophy: a long-term follow-up pilot study.
Zhao ZHANG ; Hua JIANG ; Li HUANG ; Sixi LIU ; Xiaoya ZHOU ; Yun CAI ; Ming LI ; Fei GAO ; Xiaoting LIANG ; Kam-Sze TSANG ; Guangfu CHEN ; Chui-Yan MA ; Yuet-Hung CHAI ; Hongsheng LIU ; Chen YANG ; Mo YANG ; Xiaoling ZHANG ; Shuo HAN ; Xin DU ; Ling CHEN ; Wuh-Liang HWU ; Jiacai ZHUO ; Qizhou LIAN
Protein & Cell 2025;16(1):16-27
Metachromatic leukodystrophy (MLD) is an inherited disease caused by a deficiency of the enzyme arylsulfatase A (ARSA). Lentivirus-modified autologous hematopoietic stem cell gene therapy (HSCGT) has recently been approved for clinical use in pre and early symptomatic children with MLD to increase ARSA activity. Unfortunately, this advanced therapy is not available for most patients with MLD who have progressed to more advanced symptomatic stages at diagnosis. Patients with late-onset juvenile MLD typically present with a slower neurological progression of symptoms and represent a significant burden to the economy and healthcare system, whereas those with early onset infantile MLD die within a few years of symptom onset. We conducted a pilot study to determine the safety and benefit of HSCGT in patients with postsymptomatic juvenile MLD and report preliminary results. The safety profile of HSCGT was favorable in this long-term follow-up over 9 years. The most common adverse events (AEs) within 2 months of HSCGT were related to busulfan conditioning, and all AEs resolved. No HSCGT-related AEs and no evidence of distorted hematopoietic differentiation during long-term follow-up for up to 9.6 years. Importantly, to date, patients have maintained remarkably improved ARSA activity with a stable disease state, including increased Functional Independence Measure (FIM) score and decreased magnetic resonance imaging (MRI) lesion score. This long-term follow-up pilot study suggests that HSCGT is safe and provides clinical benefit to patients with postsymptomatic juvenile MLD.
Humans
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Leukodystrophy, Metachromatic/genetics*
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Pilot Projects
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Genetic Therapy/methods*
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Hematopoietic Stem Cell Transplantation
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Male
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Follow-Up Studies
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Female
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Lentivirus/genetics*
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Child
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Child, Preschool
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Hematopoietic Stem Cells/metabolism*
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Cerebroside-Sulfatase/metabolism*
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Adolescent
2.International clinical practice guideline on the use of traditional Chinese medicine for functional dyspepsia (2025).
Sheng-Sheng ZHANG ; Lu-Qing ZHAO ; Xiao-Hua HOU ; Zhao-Xiang BIAN ; Jian-Hua ZHENG ; Hai-He TIAN ; Guan-Hu YANG ; Won-Sook HONG ; Yu-Ying HE ; Li LIU ; Hong SHEN ; Yan-Ping LI ; Sheng XIE ; Jin SHU ; Bin-Fang ZENG ; Jun-Xiang LI ; Zhen LIU ; Zheng-Hua XIAO ; Jing-Dong XIAO ; Pei-Yong ZHENG ; Shao-Gang HUANG ; Sheng-Liang CHEN ; Gui-Jun FEI
Journal of Integrative Medicine 2025;23(5):502-518
Functional dyspepsia (FD), characterized by persistent or recurrent dyspeptic symptoms without identifiable organic, systemic or metabolic causes, is an increasingly recognized global health issue. The objective of this guideline is to equip clinicians and nursing professionals with evidence-based strategies for the management and treatment of adult patients with FD using traditional Chinese medicine (TCM). The Guideline Development Group consulted existing TCM consensus documents on FD and convened a panel of 35 clinicians to generate initial clinical queries. To address these queries, a systematic literature search was conducted across PubMed, EMBASE, the Cochrane Library, China National Knowledge Infrastructure (CNKI), VIP Database, China Biology Medicine (SinoMed) Database, Wanfang Database, Traditional Medicine Research Data Expanded (TMRDE), and the Traditional Chinese Medical Literature Analysis and Retrieval System (TCMLARS). The evidence from the literature was critically appraised using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach. The strength of the recommendations was ascertained through a consensus-building process involving TCM and allopathic medicine experts, methodologists, pharmacologists, nursing specialists, and health economists, leveraging their collective expertise and empirical knowledge. The guideline comprises a total of 43 evidence-informed recommendations that span a range of clinical aspects, including the pathogenesis according to TCM, diagnostic approaches, therapeutic interventions, efficacy assessments, and prognostic considerations. Please cite this article as: Zhang SS, Zhao LQ, Hou XH, Bian ZX, Zheng JH, Tian HH, Yang GH, Hong WS, He YY, Liu L, Shen H, Li YP, Xie S, Shu J, Zeng BF, Li JX, Liu Z, Xiao ZH, Xiao JD, Zheng PY, Huang SG, Chen SL, Fei GJ. International clinical practice guideline on the use of traditional Chinese medicine for functional dyspepsia (2025). J Integr Med. 2025; 23(5):502-518.
Dyspepsia/drug therapy*
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Humans
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Medicine, Chinese Traditional/methods*
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Practice Guidelines as Topic
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Drugs, Chinese Herbal/therapeutic use*
3.Association of Body Mass Index with All-Cause Mortality and Cause-Specific Mortality in Rural China: 10-Year Follow-up of a Population-Based Multicenter Prospective Study.
Juan Juan HUANG ; Yuan Zhi DI ; Ling Yu SHEN ; Jian Guo LIANG ; Jiang DU ; Xue Fang CAO ; Wei Tao DUAN ; Ai Wei HE ; Jun LIANG ; Li Mei ZHU ; Zi Sen LIU ; Fang LIU ; Shu Min YANG ; Zu Hui XU ; Cheng CHEN ; Bin ZHANG ; Jiao Xia YAN ; Yan Chun LIANG ; Rong LIU ; Tao ZHU ; Hong Zhi LI ; Fei SHEN ; Bo Xuan FENG ; Yi Jun HE ; Zi Han LI ; Ya Qi ZHAO ; Tong Lei GUO ; Li Qiong BAI ; Wei LU ; Qi JIN ; Lei GAO ; He Nan XIN
Biomedical and Environmental Sciences 2025;38(10):1179-1193
OBJECTIVE:
This study aimed to explore the association between body mass index (BMI) and mortality based on the 10-year population-based multicenter prospective study.
METHODS:
A general population-based multicenter prospective study was conducted at four sites in rural China between 2013 and 2023. Multivariate Cox proportional hazards models and restricted cubic spline analyses were used to assess the association between BMI and mortality. Stratified analyses were performed based on the individual characteristics of the participants.
RESULTS:
Overall, 19,107 participants with a sum of 163,095 person-years were included and 1,910 participants died. The underweight (< 18.5 kg/m 2) presented an increase in all-cause mortality (adjusted hazards ratio [ aHR] = 2.00, 95% confidence interval [ CI]: 1.66-2.41), while overweight (≥ 24.0 to < 28.0 kg/m 2) and obesity (≥ 28.0 kg/m 2) presented a decrease with an aHR of 0.61 (95% CI: 0.52-0.73) and 0.51 (95% CI: 0.37-0.70), respectively. Overweight ( aHR = 0.76, 95% CI: 0.67-0.86) and mild obesity ( aHR = 0.72, 95% CI: 0.59-0.87) had a positive impact on mortality in people older than 60 years. All-cause mortality decreased rapidly until reaching a BMI of 25.7 kg/m 2 ( aHR = 0.95, 95% CI: 0.92-0.98) and increased slightly above that value, indicating a U-shaped association. The beneficial impact of being overweight on mortality was robust in most subgroups and sensitivity analyses.
CONCLUSION
This study provides additional evidence that overweight and mild obesity may be inversely related to the risk of death in individuals older than 60 years. Therefore, it is essential to consider age differences when formulating health and weight management strategies.
Humans
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Body Mass Index
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China/epidemiology*
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Male
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Female
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Middle Aged
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Prospective Studies
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Rural Population/statistics & numerical data*
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Aged
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Follow-Up Studies
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Adult
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Mortality
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Cause of Death
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Obesity/mortality*
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Overweight/mortality*
4.Research progress in targeted and immunotherapy for esophageal cancer
Zhen HUANG ; Fei YAN ; Yanling MA ; Jianhai SUN
Journal of International Oncology 2025;52(1):53-59
The early diagnosis rate of esophageal cancer is low, and most of the patients are already in the middle or late stage when diagnosed, making treatment difficult, shortening the survival period and resulting in an extremely poor prognosis. In recent years, the continuous development of targeted therapy and immunotherapy has significantly improved the survival rate and therapeutic effect of esophageal cancer patients. Analyzing the research progress of targeted therapy and immunotherapy for esophageal cancer is of great significance and can provide reference for guiding the treatment of esophageal cancer.
5.Effect of different leukocyte filters on filtration of erythrocyte suspensions
Heshan TANG ; Yan ZANG ; Zhanshan ZHA ; Weihua HUANG ; Jinqi LI ; Baohua QIAN ; Fei GUO
Journal of Navy Medicine 2025;46(9):911-917
Objective To compare the filtration effects of different models of leukocyte filters on erythrocyte suspensions,so as to provide a reference for the selection of leukocyte filters in clinic.Methods The erythrocyte suspensions prepared by Department of Blood Transfusion of The First Affiliated Hospital of Naval Medical University were used for filtration.The test was categorized into three groups based on the model of leukocyte filters,namely,AKTT-type(group Ⅰ),STTB-type(group Ⅱ),and STTA-type(groupⅢ).Each group was randomly assigned 8 bags of erythrocyte suspensions(specification 2U)with hematocrit≤55%and 10 bags of erythrocyte suspensions(specification 2U)with hematocrit>55%,and leukapheresis was applied.The quality indexes of the blood were detected before and after filtration,and the experimental data were comprehensively analyzed to evaluate the leukocyte filtration effect of various filters.Results When the hematocrit of the filtered erythrocyte suspensions was≤55%,there were significant differences in the platelet count after filtration(F=49.94,P<0.001)and filtration time(F=73.45,P<0.001)between groups,and the two indexes in group Ⅰ were superior to those in groups Ⅱ and Ⅲ.When the hematocrit of the filtered erythrocyte suspensions was>55%,there were significant differences in the platelet count after filtration(F=160.69,P<0.000 1),filtration time(F=366.09,P<0.000 1),residual leukocytes(F=4.28,P<0.05),and hemolytic rate(F=8.16,P<0.01)between groups.The platelet count after filtration and filtration time in group I were superior to those in group II and III.The indexes of residual leukocyte and hemolytic rate in groups I and II were superior to those in group III.Conclusion In order to ensure the safety and effectiveness of erythrocyte suspension transfusion,AKTT-type filter can be chosen to perform leukocyte filtration,which can further lower the blood transfusion complications.
6.Research on software development and smart manufacturing platform incorporating near-infrared spectroscopy for measuring traditional Chinese medicine manufacturing process.
Yan-Fei WU ; Hui XU ; Kai-Yi WANG ; Hui-Min FENG ; Xiao-Yi LIU ; Nan LI ; Zhi-Jian ZHONG ; Ze-Xiu ZHANG ; Zhi-Sheng WU
China Journal of Chinese Materia Medica 2025;50(9):2324-2333
Process analytical technology(PAT) is a key means for digital transformation and upgrading of the traditional Chinese medicine(TCM) manufacturing process, serving as an important guarantee for consistent and controllable TCM product quality. Near-infrared(NIR) spectroscopy has become the core technology for measuring the TCM manufacturing process. By incorporating NIR spectroscopy into PAT and starting from the construction of a smart platform for the TCM manufacturing process, this paper systematically described the development history and innovative application of the combination of NIR spectroscopy with chemometrics in measuring the TCM manufacturing process by the research team over the past two decades. Additionally, it explored the application of a validation method based on accuracy profile(AP) in the practice of NIR spectroscopy. Furthermore, the software development progress driven by NIR spectroscopy supported by modeling technology was analyzed, and the prospect of integrating NIR spectroscopy in smart factory control platforms was exemplified with the construction practices of related platforms. By integrating with the smart platform, NIR spectroscopy could improve production efficiency and guarantee product quality. Finally, the prospect of the smart platform application in measuring the TCM manufacturing process was projected. It is believed that the software development for NIR spectroscopy and the smart manufacturing platform will provide strong technical support for TCM digitalization and industrialization.
Spectroscopy, Near-Infrared/methods*
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Drugs, Chinese Herbal/analysis*
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Software
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Medicine, Chinese Traditional
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Quality Control
7.Scientific research capability of the administrative office of the Center for Disease Control and Prevention
MO Fei ; LIU Qianqiu ; LI Jiaxuan ; ZHANG Ziwei ; WANG Zhe ; HUANG Lieyu ; GUO Yan
Journal of Preventive Medicine 2025;37(12):1292-1296
Objective:
To investigate the scientific research capacity building of administrative offices of Centers for Disease Control and Prevention (CDCs) across 31 provinces (autonomous regions, municipalities), the Xinjiang Production and Construction Corps, and 5 separately listed cities in China, so as to provide the reference for improving the positioning of office functions and promoting the enhancement of scientific research capabilities.
Methods:
A self-administered questionnaire survey was conducted among heads and staff members of administrative offices in 37 CDCs. Data on office setup, general information, staffing, scientific research incentive measures and outputs were collected and analyzed.
Results:
The 37 administrative offices of the CDCs had an average authorized staffing size of 12 personnel. There were 17 of them setting independently allocated budgets. A total of 511 staff members were surveyed, comprising 238 males and 273 females, resulting in a male-to-female ratio of 0.87∶1. In terms of educational attainment, the majority held bachelor's degrees (225 individuals, 44.03%) or master's degrees and above (157 individuals, 30.72%). Professional technical personnel constituted the main occupational category, 302 individuals accounting for 59.10%. Intermediate professional titles were most common, 138 individuals accounting for 27.00%. From 2021 to 2023, a total of 68 research incentive measures have been implemented, and 579 personnel have received further training. These offices cumulatively led or participated in 80 scientific research projects and published 253 papers. Sixteen offices reported 10 and above scientific research outputs. These offices generally exhibited higher proportions of independently allocated budgets, greater numbers of senior professional titles, more staff with master's degrees or above, more implemented research incentive measures, and higher frequencies of staff further trainings.
Conclusions
The staff in the administrative offices of CDCs generally have a high level of educational attainment and include a significant number of professional technical personnel. However, their scientific research capacity remains relatively underdeveloped. It is recommended to conduct targeted professional training and research-focused lectures to enhance research literacy, leverage the strengths of multidisciplinary backgrounds, and promote cross-departmental and cross-institutional scientific research activities.
8.Highly Sensitive Detection of Water in Organic Solvents Using Pyrene-Phenol-based Fluorescent Probe
Jing LIANG ; Yan-Li WANG ; Cui-Wen JIANG ; Xiao-Chuan HUANG ; Li TANG ; Tao LI ; Yu YA ; De-Jiao NING ; Li-Ping XIE ; Fei-Yan YAN
Chinese Journal of Analytical Chemistry 2025;53(6):894-902,中插1-中插5
A pyrene-phenol-based fluorescent probe PyP which showed typical intramolecular charge transfer(ICT)and monomer-excimer activities was synthesized by using pyrene carboxaldehyde hydrazone and 4-tert-butyl-2,6-diformylphenol as the raw materials.The effects of solvents on PyP were studied,and the results showed that the color of protic polar solvents(Ethanol,N,N-dimethylformamide,methanol and H2O)were successfully identified.Based on the solvent polarity-regulated PyP monomer-excimer switching,the rapid and highly sensitive ratiometric probe,"Turn-off"and"Turn-on"multimodal probes were established for detection of trace water content in organic solvents(Dimethyl sulfoxide,N,N-dimethylformamide,ethanol and methanol),with detection limits(3σ/k)of 0.0021%,0.046%,0.062%and 0.024%.The method was successfully used to detect water content in dimethyl sulfoxide,N,N-dimethy lformamide,ethanol and methanol commercial organic solvents,with recoveries ranging from 97.2%to 108.0%.The developed method showed good accuracy and stability,and had good application prospect.
9.The Mechanism of Calcium Handling Proteins and NF-κB in Calcium Dyshomeo-stasis of Cardiomyocytes Caused by Acute MDMA Exposure
Rong-Shuai WANG ; Si-Zhe HUANG ; Yun-Yun WANG ; Yan-Fei DENG ; Zi-Jiao DING ; Jie ZHANG ; Yong LIU ; Liang REN ; Liang LIU
Journal of Forensic Medicine 2025;41(2):144-151,159
Objective To explore the mechanism of myocardial toxicity caused by N-methyl-3,4-methyle-nedioxyamphetamine(MDMA),the changes of intracellular calcium oscillation mode and calcium han-dling proteins during acute exposure to different concentrations of MDMA were detected,and the in-volvement of nuclear factor κB(NF-κB)and its effect on calcium handling proteins were investigated.Methods Primary rat cardiomyocytes were cultured to establish MDMA acute exposure model,and a control group was set up.The MDMA poisoning model was divided into three concentration groups of 10,100 and 1 000 μmol/L.After 1 h of exposure,the morphological changes of cardiomyocytes were ob-served,the cytotoxicity and changes in calcium signals were measured,and the changes in calcium handling proteins RyR2,SERCA2a,PLN,NCX1 and Cav1.2 were detected.The changes of NF-κB activity and the expression of nucleoprotein p-p65(Ser311)and PKCζ after MDMA exposure,and the intervention of NF-κB inhibitors pyrrolidine dithiocarbamate ammonium(PDTC)and protein kinase C(PKC)inhibitor chelerythrine(CHE)were detected by electrophoretic mobility shift assay(EMSA)and Western blotting.The effects of PDTC intervention on calcium signals,and the expressions of RyR2,SERCA2a,PLN,NCX1 and Cav1.2 after acute MDMA exposure were also observed.Results No obvious changes were observed in the morphology of cardiomyocytes after acute exposure to MDMA,whereas the oscillation waveform of intracytoplasmic calcium ion showed irregular changes with increased oscillation amplitude,intense fluctuations,irregular frequency,and increased fluctuation range of relative optical density values.The expression of RyR2,SERCA2a and NCX1 increased,while the expression of Cav1.2 and PLN de-creased.Acute MDMA exposure could increase NF-κB activity,while PDTC and CHE intervention could inhibit NF-κB activity.In MDMA exposed group,the expression of PKCζ and nucleoprotein p-p65(Ser311)both increased and could be inhibited by CHE.After the intervention of PDTC to block NF-κB,the amplitude of calcium oscillation was lower than that of the MDMA exposed group,and the expres-sion of RyR2,SERCA2a and NCX1 decreased.There was no significant change in PLN,while the ex-pression of Cav1.2 increased.Conclusion MDMA can lead to an increase of calcium ion concentration in cardiomyocytes.Calcium ions are involved in myocardial toxicity of MDMA.The mechanism is re-lated to changes in calcium handling proteins,mainly associated with the increased expression of RyR2.MDMA can up-regulate the intracellular activity of NF-κB through the PKCζ-NF-κB pathway and affect calcium handling proteins,which aggravate intracellular calcium overload during acute MDMA exposure.
10.Comparative analysis of characteristics and functions of exosomes from human induced pluripotent stem cell-derived platelets and apheresis platelets
Weihua HUANG ; Yan ZANG ; Aihua QIN ; Ziyang FENG ; Heshan TANG ; Fei GUO ; Chuyan WU ; Qiu SHEN ; Baohua QIAN ; Haihui GU ; Zhanshan CHA
Chinese Journal of Blood Transfusion 2025;38(9):1154-1161
Objective: To compare the biological characteristics of human induced pluripotent stem cell-derived platelet exosomes (hiPSC-Plt-Exos) with those of conventional apheresis platelet exosomes (Plt-Exos), specifically focusing on their differential abilities to enhance the proliferation and migration of human umbilical cord mesenchymal stem cells (hUC-MSCs). Methods: Exosomes were isolated from hiPSC-derived Plt and apheresis Plt concentrate using size exclusion chromatography. These exosomes were then characterized through nanoparticle tracking analysis (NTA), transmission electron microscopy (TEM), and Western blotting. Co-culture experiments into hUC-MSCs were conducted with hiPSC-Plt-Exos and apheresis Plt-Exos, respectively. Their effects on the proliferation and migration of hUC-MSCs were assessed via cell proliferation assays and scratch tests. Results: hiPSC-Plt-Exos and apheresis Plt-Exos exhibited comparable particle sizes, morphological features (such as the characteristic cup-shaped structure), and surface markers (including CD9 and HSP70). Notably, hiPSC-Plt-Exos demonstrated a significantly greater ability to enhance the proliferation and migration of hUC-MSCs compared to apheresis Plt-Exos (P<0.05). These differences provide critical comparative data for their application in various clinical contexts. Conclusion: This study establishes a theoretical foundation for developing precise therapeutic strategies based on hiPSC-Plt-Exos. Furthermore, it underscores the necessity of selecting the appropriate type of exosomes according to the specific disease microenvironment to achieve optimal therapeutic outcomes.


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