1.A multi-omics-empowered framework for precision diagnosis and treatment of lysosomal diseases.
Nguyen Thi Hai YEN ; Nguyen Tran Nam TIEN ; Nguyen Quang THU ; Franklin DUCATEZ ; Wladimir MAUHIN ; Olivier LIDOVE ; Soumeya BEKRI ; Abdellah TEBANI ; Nguyen Phuoc LONG
Journal of Pharmaceutical Analysis 2025;15(10):101274-101274
Lysosomal diseases (LDs) are a group of rare inherited disorders belonging to inborn metabolism errors. LDs are characterized by the excessive storage of undegraded substrates, most often due to the enzymatic deficiency resulting from disease-causing gene variants. LDs lead to dysregulated cellular pathways and imbalanced molecular homeostasis and can affect multiple organs and tissues. Despite being rare, LDs account for a significant incidence when considered collectively. Due to complex molecular and genetic fingerprints, considerable challenges in LD management must be overcome. Diagnosis can be significantly delayed due to the broad and nonspecific clinical manifestations and the lack of specific biomarkers. Available treatments fail to fully stop the disease progression and can alter the disease's typical phenotypes with novel manifestations. Therefore, a paradigm shift is crucial to better understand LDs and provide actionable insights. Herein, we comprehensively review the literature to demonstrate that multi-omics approaches are promising for pathophysiology elucidation, biomarker discovery, and precision therapy in LDs. We recommend adopting longitudinal study designs integrated with a multi-omics-empowered framework to facilitate mechanistic delineation, biomarker discovery, and treatment development. Relevant approaches exploring the association between LDs and common neurodegenerative disorders are also discussed, paving a potential path for improved therapeutic development and ultimately improving the patient's quality of life.
2.Clinical and molecular characteristics of simple virilizing congenital adrenal hyperplasia due to 21-hydroxylase deficiency: insight from a tertiary pediatric center in Vietnam
Khanh Ngoc NGUYEN ; Giang Thi Kim DANG ; Ngoc Thi Bich CAN ; Dien Minh TRAN ; Thao Phuong BUI ; Mai Nguyen Thi PHUONG ; Huong Thu PHAM ; Ngoc Diem NGO ; Dung Chi VU
Annals of Pediatric Endocrinology & Metabolism 2025;30(6):330-339
Purpose:
Simple virilizing congenital adrenal hyperplasia (SV-CAH) due to 21-hydroxylase deficiency (21-OHD) is an autosomal recessive disease caused by pathogenic variants of the CYP21A2 gene. Children with SV-CAH often experience delayed diagnosis, presenting with pseudo-precocious puberty in males and genital virilization in females. Genotyping is essential for diagnosis, treatment, optimization, and phenotype prediction. This study describes the clinical and genetic characteristics of SV-CAH to guide treatment strategies.
Methods:
From November 2016 to March 2023, 79 children (accounting for 34.3% of 230 CAH cases in the overall children’s cohort) from 75 families were classified as SV-CAH due to 21-OHD at the Vietnam National Children's Hospital. Forty-three children underwent CYP21A2 mutation analysis using multiplex ligation-dependent probe amplification and complete gene sequencing to detect pathogenic variants.
Results:
Median age at diagnosis was 4.5 years (interquartile range, 1 day–22.3 years). There were 38.0% males and 62.0% females. The most common symptoms were penile enlargement in males (53.3%) and clitoromegaly (87.8%) in females; the height standard deviation (SD) at diagnosis was 1.90±1.79 SD (-2.02 to 5.43) according to the World Health Organization; and bone age advancement was 4.65±2.59 years. Genetic analysis identified 21 pathogenic variants and 22 genotypes in 43 children. The most common variant was p.I173N (47.7%); the most common genotype was p.I173N/p.I173N (16.3%).
Conclusion
Children with SV-CAH are often diagnosed late. To avoid that, early genetic analysis should be prioritized, especially for children diagnosed through newborn screening programs. Determining the genotype is crucial for optimizing treatment strategies, ensuring personalized management, and avoiding overtreatment.
3.In Vitro Evaluation of the Anti-Proliferative and Anti-Diabetic Activities of Streptocaulon juventas Leaf Extracts
Tran Nhat MAI ; Bui Thi PHUONG ; Tran Thi HUYEN ; Nguyen Minh NAM
Natural Product Sciences 2025;31(4):278-283
Streptocaulon juventas, a medicinal plant traditionally used in Vietnam and other Southeast Asian countries, has been previously investigated for certain biological activities of its roots; however, studies on its leaves remain limited. In this study, leaf extracts were evaluated for their in vitro anti-proliferative activity against B16F10 (murine melanoma) and HepG2 (human hepatocellular carcinoma) cell lines, as well as for their αglucosidase inhibitory potential. MTT assays revealed that the hexane (Sj-Hx) and dichloromethane (Sj-DCM) fractions exhibited the strongest cytotoxic effects on B16F10 cells, with IC₅₀ values of 36.09 µg/mL and 45.91 µg/mL, respectively. The hexane fraction (Sj-Hx) also demonstrated potent activity against HepG2 cells, with an IC50 value of 36.65 µg/mL. Notably, none of the extracts exhibited cytotoxicity toward Hs68 normal human fibroblasts, indicating favorable selectivity. DAPI staining further confirmed that both Sj-Hx and Sj-DCM induced nuclear fragmentation and chromatin condensation, consistent with apoptosis induction. Additionally, in vitro assays of anti-diabetic activity revealed that the hexane (Sj-Hx) and ethyl acetate (Sj-EA) extracts displayed the strongest α-glucosidase inhibition, with IC₅₀ values of 221.59 µg/mL and 265.73 µg/mL, respectively. Collectively, these findings suggest that S. juventas leaf extracts possess selective pro-apoptotic activity against cancer cells and represent a promising natural source for the discovery of anti-diabetic agents.
4.Anti-Inflammatory and Immunoregulatory Activities In Vitro of Coumarins Isolated from the Twigs and Leaves of Wikstroemia indica (L.) C.A. Mey.
Van Lieu NGUYEN ; Van Anh CAO ; Thi Thu NGUYEN ; Thi Hien TRAN ; Viet Dung HOANG ; Thi Ha DO
Natural Product Sciences 2025;31(4):284-290
Six coumarins were isolated from two bioactive fractions of the 70% ethanol extract from the twigsand leaves of Wikstroemia indica (L.) C.A. Mey. (Thymelaeaceae), namely the ethyl acetate fraction (WIE) and the water fraction (WIW). These compounds were identified as umbelliferone (1), daphnoretin (2), daphnorin (3), adicardin (4), wikstronutin (5), and triumbelletin-7-O-β-D-glucoside (6). Compound 4 is reported for the first time in the genus Wikstroemia, whereas compound 5 is newly identified in this species. All six compounds were evaluated for their effects on Interleukin-1 beta (IL-1β) and IL-10 secretion in Lipopolysaccharide (LPS)-stimulated RAW 264.7 macrophages, among which compounds 1, 2, 5, and 6 showed stronger activity. These four coumarin derivatives further suppressed the production of Foxp3, Interferon-alpha (IFN-α), IFN-γ, and IL-17 in LPSstimulated CD4+ T cells. The IC50 values ranged from 18.84 ± 1.16 to 43.45 ± 5.34 μM for Foxp3, 8.96 ± 1.45 to 22.42 ± 4.12 μM for IFN-α, 9.28 ± 1.23 to 16.83 ± 2.15 μM for IFN-γ, and 12.62 ± 2.57 to 22.92 ± 4.41 μM for IL-17. Notably, compound 6 exhibited the strongest immunomodulatory effect. Overall, the findings suggest that coumarins from W. indica possess promising immunomodulatory and anti-inflammatory potential.
5.Sesquiterpenes from Curcuma zedoaria (Christm.) Rosc. Rhizomes and Their Alpha-Glucosidase Inhibitory Effects
Ngan Thi TRAN ; Phuong Nguyen Thi THU ; Mai-Ngan Thi LE ; Quynh-Mai Thi NGO
Natural Product Sciences 2024;30(4):300-303
Curcuma zedoaria (Christm.) Rosc. is a popular traditional herb to treat digestive disorders in Asian tropical countries. Previous studies indicated the presence of sesquiterpenoids, diterpenoids, and curcuminoids with various bioactivities. To enrich the phytocomposition data of this plant, this investigation was conducted.The dried rhizomes of C. zedoaria were collected in Hai Phong City (Vietnam), extracted with methanol and fractionated with n-hexane, CH2Cl2 , and EtOAc. Compounds were isolated from n-hexane soluble fraction by open column chromatography combined with thin layer chromatography from fraction n-hexane. Their chemical structures were elucidated by 1D, and 2D NMR spectra and comparison with reported data. As a result, a phytochemical investigation was conducted to isolate six sesquiterpenes from C. zedoaria. Their chemical structures were elucidated to be curcumenol (1), procurcumenol (2), neoprocurcumenol (3), 13-hydroxygermacrone (4), zederone (5), and curcumalactone (6). Among isolated compounds, compounds 1, 2, 4, and 5 were reported from C. zedoaria. Meanwhile, neoprocurcumenol (3) and curcumalactone (6) are isolated from this species for the first time. Compound 5 exhibited a mild inhibitory effect on α-glucosidase with an IC50 of 99.45 ± 0.50 μg/mL.
6.Push forward LC-MS-based therapeutic drug monitoring and pharmacometabolomics for anti-tuberculosis precision dosing and comprehensive clinical management.
Nguyen Quang THU ; Nguyen Tran Nam TIEN ; Nguyen Thi Hai YEN ; Thuc-Huy DUONG ; Nguyen Phuoc LONG ; Huy Truong NGUYEN
Journal of Pharmaceutical Analysis 2024;14(1):16-38
The spread of tuberculosis (TB), especially multidrug-resistant TB and extensively drug-resistant TB, has strongly motivated the research and development of new anti-TB drugs. New strategies to facilitate drug combinations, including pharmacokinetics-guided dose optimization and toxicology studies of first- and second-line anti-TB drugs have also been introduced and recommended. Liquid chromatography-mass spectrometry (LC-MS) has arguably become the gold standard in the analysis of both endo- and exo-genous compounds. This technique has been applied successfully not only for therapeutic drug monitoring (TDM) but also for pharmacometabolomics analysis. TDM improves the effectiveness of treatment, reduces adverse drug reactions, and the likelihood of drug resistance development in TB patients by determining dosage regimens that produce concentrations within the therapeutic target window. Based on TDM, the dose would be optimized individually to achieve favorable outcomes. Pharmacometabolomics is essential in generating and validating hypotheses regarding the metabolism of anti-TB drugs, aiding in the discovery of potential biomarkers for TB diagnostics, treatment monitoring, and outcome evaluation. This article highlighted the current progresses in TDM of anti-TB drugs based on LC-MS bioassay in the last two decades. Besides, we discussed the advantages and disadvantages of this technique in practical use. The pressing need for non-invasive sampling approaches and stability studies of anti-TB drugs was highlighted. Lastly, we provided perspectives on the prospects of combining LC-MS-based TDM and pharmacometabolomics with other advanced strategies (pharmacometrics, drug and vaccine developments, machine learning/artificial intelligence, among others) to encapsulate in an all-inclusive approach to improve treatment outcomes of TB patients.
7.Neuroprotective mechanism of corydaline in glutamate-induced neurotoxicity in HT22 cells
Baskar SELVARAJ ; Dae Won KIM ; Ki-Yeon YOO ; Keunwan PARK ; Thi Thu Thuy TRAN ; Jae Wook LEE ; Heesu LEE
International Journal of Oral Biology 2024;49(1):10-17
Glutamate-mediated oxidative stress causes neuronal cell death by increasing intracellular Ca2+ uptake, reactive oxidative species (ROS) generation, mitogen-activated protein kinase (MAPK) activation, and translocation of apoptosis-inducing factor (AIF) to the nucleus. In the current study, we demonstrated that corydaline exerts potent neuroprotective effects against glutamate-induced neurotoxicity. Treatment with 5 mmol/L glutamate increased cellular Ca2+ influx, ROS generation, MAPK activation, and AIF translocation. In contrast, corydaline treatment decreased cellular Ca2+ influx and ROS generation. Western blot analysis revealed that glutamate-mediated MAPK activation was attenuated by corydaline treatment. We further demonstrated that corydaline treatment inhibited the glutamate-mediated translocation of AIF to the nucleus. We propose that corydaline is a promising lead structure for the development of safe and effective neuroprotectants.
8.The effect of simulation-based training on problem-solving skills, critical thinking skills, and self-efficacy among nursing students in Vietnam: a before-and-after study
Tran Thi Hoang OANH ; Luu Thi THUY ; Ngo Thi Thu HUYEN
Journal of Educational Evaluation for Health Professions 2024;21(1):24-
Purpose:
This study investigated the effect of simulation-based training on nursing students’ problem-solving skills, critical thinking skills, and self-efficacy.
Methods:
A single-group pretest and posttest study was conducted among 173 second-year nursing students at a public university in Vietnam from May 2021 to July 2022. Each student participated in the adult nursing preclinical practice course, which utilized a moderate-fidelity simulation teaching approach. Instruments including the Personal Problem-Solving Inventory Scale, Critical Thinking Skills Questionnaire, and General Self-Efficacy Questionnaire were employed to measure participants’ problem-solving skills, critical thinking skills, and self-efficacy. Data were analyzed using descriptive statistics and the paired-sample t-test with the significance level set at P<0.05.
Results:
The mean score of the Personal Problem-Solving Inventory posttest (127.24±12.11) was lower than the pretest score (131.42±16.95), suggesting an improvement in the problem-solving skills of the participants (t172=2.55, P=0.011). There was no statistically significant difference in critical thinking skills between the pretest and posttest (P=0.854). Self-efficacy among nursing students showed a substantial increase from the pretest (27.91±5.26) to the posttest (28.71±3.81), with t172=-2.26 and P=0.025.
Conclusion
The results suggest that simulation-based training can improve problem-solving skills and increase self-efficacy among nursing students. Therefore, the integration of simulation-based training in nursing education is recommended.
9.Sesquiterpenes from Curcuma zedoaria (Christm.) Rosc. Rhizomes and Their Alpha-Glucosidase Inhibitory Effects
Ngan Thi TRAN ; Phuong Nguyen Thi THU ; Mai-Ngan Thi LE ; Quynh-Mai Thi NGO
Natural Product Sciences 2024;30(4):300-303
Curcuma zedoaria (Christm.) Rosc. is a popular traditional herb to treat digestive disorders in Asian tropical countries. Previous studies indicated the presence of sesquiterpenoids, diterpenoids, and curcuminoids with various bioactivities. To enrich the phytocomposition data of this plant, this investigation was conducted.The dried rhizomes of C. zedoaria were collected in Hai Phong City (Vietnam), extracted with methanol and fractionated with n-hexane, CH2Cl2 , and EtOAc. Compounds were isolated from n-hexane soluble fraction by open column chromatography combined with thin layer chromatography from fraction n-hexane. Their chemical structures were elucidated by 1D, and 2D NMR spectra and comparison with reported data. As a result, a phytochemical investigation was conducted to isolate six sesquiterpenes from C. zedoaria. Their chemical structures were elucidated to be curcumenol (1), procurcumenol (2), neoprocurcumenol (3), 13-hydroxygermacrone (4), zederone (5), and curcumalactone (6). Among isolated compounds, compounds 1, 2, 4, and 5 were reported from C. zedoaria. Meanwhile, neoprocurcumenol (3) and curcumalactone (6) are isolated from this species for the first time. Compound 5 exhibited a mild inhibitory effect on α-glucosidase with an IC50 of 99.45 ± 0.50 μg/mL.
10.STRESS IN RELATIONSHIP WITH BURNOUT DUE TO COVID-19: THE MODERATING ROLE OF COPING STRATEGIES
Tran Le Thanh ; Nguyen Thanh Hoang ; Nguyen Thi Mai Lan ; Vu Thu Trang ; Le Vu Ha ; Nguyen Thi Hoa Mai ; Cao Xuan Lieu ; Ha Thi Minh Chinh
ASEAN Journal of Psychiatry 2024;25(3):1-10
STRESS IN RELATIONSHIP WITH BURNOUT DUE TO COVID-19: THE MODERATING ROLE OF COPING STRATEGIES
This study investigates the relationship between stress and burnout related to the
COVID-19 pandemic, focusing on the moderating influence of positive and negative
coping strategies. In a cross-sectional survey involving 3,664 Vietnamese citizens aged 18
and above, the research explores the dynamics of stress and burnout symptoms associated
with the ongoing pandemic. The results reveal a negative impact of stress symptoms on
COVID-19-related burnout among the population. Notably, both positive and negative
coping strategies are identified as significant moderators in the interplay between stress
and burnout. This suggests that individuals’ coping mechanisms influence the severity
of stress and burnout experienced in dealing with COVID-19 challenges. These findings
contribute to the foundational understanding of the stress-burnout relationship during the
pandemic and provide essential insights for interventions. By recognizing the moderating
role of coping strategies, this study lays the groundwork for strategies aimed at enhancing
individuals’ coping abilities, ultimately reducing stress and burnout levels. These insights
hold the potential to assist individuals in adapting more effectively to the challenges posed
by the COVID-19 pandemic, fostering improved mental well-being in the face of ongoing
uncertainties. ASEAN Journal of Psychiatry, Vol. 25 (3) March, 2024; 1-10.


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