1.Increased risk of osteoporotic pathological fractures associated with sterol esters:evidence from IEU-GWAS and FinnGen databases
Zengjie GAO ; Xiang PU ; Lailai LI ; Yihui CHAI ; Hua HUANG ; Yu QIN
Chinese Journal of Tissue Engineering Research 2026;30(5):1302-1310
BACKGROUND:Although previous studies have reported associations between lipids and the risk of osteoporotic pathological fractures,the specific causal relationships between lipid level and osteoporotic pathological fractures remain unclear.OBJECTIVE:To elucidate the causal relationship between lipids and osteoporotic pathological fractures using a two-sample bidirectional Mendelian randomization analysis.METHODS:The data for 178 lipid metabolites were obtained from the IEU-GWAS database(developed by the MRC Integrative Epidemiology Unit at the University of Bristol,UK,which provides extensive summary data from genome-wide association studies),while osteoporotic pathological fracture data(from 173 619 European participants)were acquired from the FinnGen database(constructed by the Finnish national gene research program,focusing on investigating relationships between genomics and health/disease in the Finnish population).Osteoporotic pathological fracture data were used as the outcome variable,with lipids serving as exposures,for the bidirectional Mendelian randomization study to evaluate the causal effects of different lipids on osteoporotic pathological fractures.The UK Biobank database was employed as a validation set by switching the outcome variable to verify the findings horizontally.RESULTS AND CONCLUSION:(1)The inverse variance weighted analysis indicated that each unit increase in sterol ester(27∶1/20∶2)levels was associated with a 25.55%increase in the risk of osteoporotic pathological fractures(odds ratio=1.256,95%confidence interval:1.001-1.575,P=0.049),suggesting a significant positive correlation between elevated sterol ester levels and increased fracture risk.Reverse Mendelian randomization analysis revealed a significant negative association between osteoporotic pathological fractures and three types of phosphatidylcholine.Horizontal validation yielded consistent results,confirming sterol ester as a risk factor for osteoporotic pathological fractures.(2)The results indicate that sterol ester is a risk factor for osteoporotic pathological fractures,while phosphatidylcholine serves as a protective factor.These findings strengthen the evidence supporting the effect of lipids on the risk of osteoporotic pathological fractures.Although the GWAS data used in this study were derived from European populations,given the broad commonality of human genetics,the results provide valuable reference significance for improving osteoporosis in Chinese populations through lipid regulation.
2.Influencing factors of significant corneal astigmatism in pterygium patients during the perioperative period
Shiru CHAI ; Xiaofen ZHENG ; Hua YU ; Zhen LI ; Yuguo KANG
International Eye Science 2026;26(4):683-686
AIM: To explore the factors associated with significant corneal astigmatism during the perioperative period in patients with pterygium. METHODS: Patients with primary pterygium presenting at Shanxi Eye Hospital between February and June 2025 were enrolled. All patients underwent medical history collection. Pre- and postoperative data were obtained using Pentacam, anterior segment photography, Image J software, and anterior segment optical coherence tomography(AS-OCT). All patients underwent pterygium excision combined with autologous bulbar conjunctival flap transplantation under local infiltration anesthesia. RESULTS: A total of 76 patients(76 eyes)with pterygium were finally enrolled(30 males, 46 females)with a mean age of 62.2±8.2 y. The mean length of corneal invasion by pterygium was 3.61±0.89 mm, the mean depth of invasion into the anterior corneal surface was 0.15±0.09 mm, and the median area of corneal invasion was 10.25(6.90, 18.75)mm2. The median preoperative corneal astigmatism was 1.50(0.70, 5.45)D. Median astigmatism was 0.8(0.40, 1.28)D at 2 wk postoperatively and 0.60(0.30, 1.15)D at 1 mo postoperatively. Patient age showed a positive correlation with preoperative astigmatism, and with residual astigmatism at 2 wk and 1 mo postoperatively(all P<0.05). The length of corneal invasion was positively correlated with preoperative astigmatism and residual astigmatism at both postoperative timepoints(P<0.01). The depth of invasion showed no significant linear correlation with astigmatism at any stage(P=0.250, 0.761, 0.686). The area of corneal invasion was positively correlated with astigmatism at all stages(P<0.01). Patients were grouped based on significant astigmatism(≥1.0 D)and non-significant astigmatism(<1.0 D), after adjusting for other variables, age(P=0.031)and the area of corneal invasion(P=0.004)were identified as risk factors for significant astigmatism. Pterygium invasion length was not significant factors(P>0.05). Receiver operating characteristic(ROC)analysis showed the highest area under the curve(AUC)for the invasion area(AUC=0.915). CONCLUSION: Significant preoperative corneal astigmatism in pterygium patients is correlated with patient age, the length of corneal invasion, and the area of invasion. The area of pterygium invasion into the cornea is the strongest predictor of significant preoperative corneal astigmatism.
3.Increased risk of osteoporotic pathological fractures associated with sterol esters:evidence from IEU-GWAS and FinnGen databases
Zengjie GAO ; Xiang PU ; Lailai LI ; Yihui CHAI ; Hua HUANG ; Yu QIN
Chinese Journal of Tissue Engineering Research 2026;30(5):1302-1310
BACKGROUND:Although previous studies have reported associations between lipids and the risk of osteoporotic pathological fractures,the specific causal relationships between lipid level and osteoporotic pathological fractures remain unclear.OBJECTIVE:To elucidate the causal relationship between lipids and osteoporotic pathological fractures using a two-sample bidirectional Mendelian randomization analysis.METHODS:The data for 178 lipid metabolites were obtained from the IEU-GWAS database(developed by the MRC Integrative Epidemiology Unit at the University of Bristol,UK,which provides extensive summary data from genome-wide association studies),while osteoporotic pathological fracture data(from 173 619 European participants)were acquired from the FinnGen database(constructed by the Finnish national gene research program,focusing on investigating relationships between genomics and health/disease in the Finnish population).Osteoporotic pathological fracture data were used as the outcome variable,with lipids serving as exposures,for the bidirectional Mendelian randomization study to evaluate the causal effects of different lipids on osteoporotic pathological fractures.The UK Biobank database was employed as a validation set by switching the outcome variable to verify the findings horizontally.RESULTS AND CONCLUSION:(1)The inverse variance weighted analysis indicated that each unit increase in sterol ester(27∶1/20∶2)levels was associated with a 25.55%increase in the risk of osteoporotic pathological fractures(odds ratio=1.256,95%confidence interval:1.001-1.575,P=0.049),suggesting a significant positive correlation between elevated sterol ester levels and increased fracture risk.Reverse Mendelian randomization analysis revealed a significant negative association between osteoporotic pathological fractures and three types of phosphatidylcholine.Horizontal validation yielded consistent results,confirming sterol ester as a risk factor for osteoporotic pathological fractures.(2)The results indicate that sterol ester is a risk factor for osteoporotic pathological fractures,while phosphatidylcholine serves as a protective factor.These findings strengthen the evidence supporting the effect of lipids on the risk of osteoporotic pathological fractures.Although the GWAS data used in this study were derived from European populations,given the broad commonality of human genetics,the results provide valuable reference significance for improving osteoporosis in Chinese populations through lipid regulation.
4.Observation on the effect of esketamine combined with general anesthesia and ultrasound-guided cervical plexus block in axillary endoscopic thyroidectomy
Rui LI ; Weifeng YAN ; Shengyang DENG ; Hua CHAI ; Fei FENG
China Journal of Endoscopy 2025;31(10):30-39
Objective To investigate the effect and safety of esketamine combined with general anesthesia and ultrasound-guided cervical plexus block in axillary endoscopic thyroidectomy.Methods The subjects of this study,112 female patients who underwent axillary endoscopic thyroidectomy in our hospital from January 2022 to October 2024,were randomly divided into an observation group and a control group,each with 56 cases.All patients were administered with ultrasound-guided cervical plexus block followed by general anesthesia.The observation group was injected with 0.1 mg/kg of esketamine prior to anesthesia induction and continuously pumped with esketamine at a rate of 0.1 mg/(kg·h)during the operation until sutures were completed,while the control group was administered with an equal volume of normal saline following the same procedure.The vital sign indicators[mean arterial pressure(MAP),heart rate(HR)and percutaneous arterial oxygen saturation(SpO2)]immediately after establishing electrocardiogram monitoring(T0),immediately before tracheal intubation(T1),immediately after tracheal intubation(T2),immediately after skin incision(T3)and immediately after the operation(T4)were compared between the two groups.Surgical related indicators such as propofol dosage,remifentanil dosage,and extubation time were also compared between them.Pain visual analogue scale(VAS)scores were performed on patients from the two groups at 1,6,12,and 24 h postoperatively.The 40-item quality of recovery score(QoR-40)was used before the surgeries and 1 d after surgeries to assess the patients.Adverse reactions were also compared.Results The difference was significant in terms of MAP,HR time effect in the two groups(F=24.63,F=20.11,P<0.05)and inter-group MAP,HR values(F=13.54,F=11.49,P<0.05).There were statistically significant differences in the interaction effects of MAP and HR between the two groups(F=4.10,F=3.46,P<0.05).The difference was not statistically significant in time,inter group,and interaction effects of SpO2(P>0.05).The dosage of propofol,remifentanil,and postoperative 1 d drainage in the observation group were less than those in the control group,the differences were statistically significant(P<0.05).The differences were not statistically signiciant in extubation time and awakening time between the two groups(P>0.05).The VAS scores at 1,6,12,and 24 h after operation in the observation groups were lower than those in the control group,the differences were statistically significant(P<0.05).Physical comfort,pain dimension ratings,and total score of the QoR-40 scale:1)those 1 d after operation were lower than those before operation in both groups(P<0.05);2)1 d after operation:those in the control group were lower than those in the observation group(P<0.05).The control group witnessed a remarkable decrease in QoR-40 emotional state rating 1 d after operation than it did before operation(P<0.05),while no significant change was seen by the observation group in this regard(P>0.05).The incidence of coughing after extubation in the observation group was lower than that in the control group(14.29%and 37.50%),the difference was statistically significant(P<0.05).The differences were not statistically signiciant in the incidence of other adverse reactions such as nausea,vomiting,dizziness,headache,respiratory depression,awakening restlessness and awakening delirium between the two groups(P>0.05).The total incidence of adverse reactions in the observation group was significantly lower than that in the control group(25.00%and 66.07%,P<0.05).Conclusion The application of esketamine combined with general anesthesia and ultrasound-guided cervical plexus block in axillary endoscopic thyroidectomy can significantly stabilize hemodynamics in patients,reduce postoperative pain,and promote postoperative recovery.It is worthy clinical application.
5.Effect of PHC combined with RIPC on postoperative cognitive function in patients with lung cancer
Hua CHAI ; Yulong DU ; Wenyong PENG
China Modern Doctor 2025;63(4):6-10
Objective To investigate the effects of permissive hypercapnia(PHC)combined with remote ischemic preconditioning(RIPC)on regional brain oxygen saturation(rSO2)and postoperative cognitive function in patients undergoing thoracoscopic radical resection for lung cancer.Methods A total of 80 patients who underwent thoracoscopic lung cancer resection in Jinhua Municipal Central Hospital from January to December 2022 were selected and divided into control group,RIPC group,PHC group and PHC+RIPC group according to random number table method,with 20 cases in each group.rSO2 levels were observed before anesthesia induction(T0),before unilateral ventilation(T1),10min after unilateral ventilation(T2),30min after unilateral ventilation(T3),at the end of after unilateral ventilation(T4)and after surgery(T5)in four groups.The level of serum interleukin(IL)-6,IL-10,tumor necrosis factor-α(TNF-α)and cognitive function at T0,T4,the first day after surgery(T6)and the third day postoperative(T7)were recorded.Results After surgery,the levels of IL-6,TNF-α,and IL-10 in four groups first increased and then decreased(P<0.05).At T4 and T6,there were statistically significant differences in IL-6 levels among four groups(P<0.05),and the IL-6 level of control group was significantly higher than that of other three groups(P<0.05).At T4,T6 and T7,there were statistically significant differences in IL-10 and TNF-α levels among four groups(P<0.05),and the IL-10 level in control group was significantly lower than that in other three groups,and the TNF-α level was significantly higher than that in other three groups.At T2,T3,T4 and T5,there were statistically significant differences in rSO2 between four groups(P<0.05),and PHC+RIPC group>RIPC group>PHC group>control group.At T6,the score of mini-mental state examination scale of control group was significantly lower than that of other three groups(P<0.05).Conclusion PHC combined with RIPC can improve rSO2 and postoperative cognitive function in patients with thoracoscopic lung cancer resection,reduce the level of inflammatory factors,and reduce the occurrence of postoperative cognitive dysfunction.
6.Observation on the effect of esketamine combined with general anesthesia and ultrasound-guided cervical plexus block in axillary endoscopic thyroidectomy
Rui LI ; Weifeng YAN ; Shengyang DENG ; Hua CHAI ; Fei FENG
China Journal of Endoscopy 2025;31(10):30-39
Objective To investigate the effect and safety of esketamine combined with general anesthesia and ultrasound-guided cervical plexus block in axillary endoscopic thyroidectomy.Methods The subjects of this study,112 female patients who underwent axillary endoscopic thyroidectomy in our hospital from January 2022 to October 2024,were randomly divided into an observation group and a control group,each with 56 cases.All patients were administered with ultrasound-guided cervical plexus block followed by general anesthesia.The observation group was injected with 0.1 mg/kg of esketamine prior to anesthesia induction and continuously pumped with esketamine at a rate of 0.1 mg/(kg·h)during the operation until sutures were completed,while the control group was administered with an equal volume of normal saline following the same procedure.The vital sign indicators[mean arterial pressure(MAP),heart rate(HR)and percutaneous arterial oxygen saturation(SpO2)]immediately after establishing electrocardiogram monitoring(T0),immediately before tracheal intubation(T1),immediately after tracheal intubation(T2),immediately after skin incision(T3)and immediately after the operation(T4)were compared between the two groups.Surgical related indicators such as propofol dosage,remifentanil dosage,and extubation time were also compared between them.Pain visual analogue scale(VAS)scores were performed on patients from the two groups at 1,6,12,and 24 h postoperatively.The 40-item quality of recovery score(QoR-40)was used before the surgeries and 1 d after surgeries to assess the patients.Adverse reactions were also compared.Results The difference was significant in terms of MAP,HR time effect in the two groups(F=24.63,F=20.11,P<0.05)and inter-group MAP,HR values(F=13.54,F=11.49,P<0.05).There were statistically significant differences in the interaction effects of MAP and HR between the two groups(F=4.10,F=3.46,P<0.05).The difference was not statistically significant in time,inter group,and interaction effects of SpO2(P>0.05).The dosage of propofol,remifentanil,and postoperative 1 d drainage in the observation group were less than those in the control group,the differences were statistically significant(P<0.05).The differences were not statistically signiciant in extubation time and awakening time between the two groups(P>0.05).The VAS scores at 1,6,12,and 24 h after operation in the observation groups were lower than those in the control group,the differences were statistically significant(P<0.05).Physical comfort,pain dimension ratings,and total score of the QoR-40 scale:1)those 1 d after operation were lower than those before operation in both groups(P<0.05);2)1 d after operation:those in the control group were lower than those in the observation group(P<0.05).The control group witnessed a remarkable decrease in QoR-40 emotional state rating 1 d after operation than it did before operation(P<0.05),while no significant change was seen by the observation group in this regard(P>0.05).The incidence of coughing after extubation in the observation group was lower than that in the control group(14.29%and 37.50%),the difference was statistically significant(P<0.05).The differences were not statistically signiciant in the incidence of other adverse reactions such as nausea,vomiting,dizziness,headache,respiratory depression,awakening restlessness and awakening delirium between the two groups(P>0.05).The total incidence of adverse reactions in the observation group was significantly lower than that in the control group(25.00%and 66.07%,P<0.05).Conclusion The application of esketamine combined with general anesthesia and ultrasound-guided cervical plexus block in axillary endoscopic thyroidectomy can significantly stabilize hemodynamics in patients,reduce postoperative pain,and promote postoperative recovery.It is worthy clinical application.
7.Evaluating the effectiveness of immediate vs. elective thoracic endovascular aortic repair for blunt thoracic aortic injury.
Zhaohui HUA ; Baoning ZHOU ; Wenhao XUE ; Zhibin ZHOU ; Jintao SHAN ; Lei XIA ; Yunpeng LUO ; Yiming CHAI ; Zhen LI
Chinese Journal of Traumatology 2025;28(1):22-28
PURPOSE:
To evaluate the relationship between the timing of thoracic endovascular aortic repair (TEVAR) for blunt thoracic aortic injury (BTAI) and prognosis.
METHODS:
This is a single-center retrospective cohort study. Patients who received TEVAR for BTAI at our institution from October 2016 to September 2023 were divided into 2 categories depending on the injury severity score (ISS) (≤ 25 vs. > 25) and when the TEVAR was performed for BTAI (within 24 h vs. after 24 h), respectively. The analysis included all patients who received TEVAR treatment after being diagnosed with BTAI through whole-body CT angiography. Patients treated with open repair and non-operative management were excluded. After propensity-score matching for various factors, outcomes during hospitalization and follow-up were compared. These factors included demographics, comorbidities, concomitant injuries, cause and location of aortic injury, Glasgow coma scale score, society for vascular surgery grading, hemoglobin concentration, creatinine concentration, shock, systolic blood pressure, and heart rate at admission. The comparison was conducted using SPSS 26 software. Continuous variables were presented as either the mean ± standard deviation or median (Q1, Q3), and were compared using either the t-test or the Mann-Whitney U test. Categorical variables were expressed as n (%), and comparisons were made between the 2 groups using the χ2 test or Fisher's exact test. Statistical significance was defined as a 2-sided p < 0.05.
RESULTS:
In total, 110 patients were involved in the study, with 65 (59.1%) patients having ISS scores > 25 and 32 (29.1%) receiving immediate TEVAR. The perioperative overall mortality rate in the group with ISS > 25 was significantly higher than that in the group with ISS ≤ 25 (11 (16.9%) vs. 2 (4.4%), p < 0.001). Upon admission, the elective group exhibited a notably higher Glasgow coma scale score (median (Q1, Q3)) compared to the immediate group (15 (12, 15) vs. 13.5 (9, 15), p = 0.039), while the creatinine concentration (median (Q1, Q3)) at admission was significantly higher in the immediate group (90.5 (63.8, 144.0) vs. 71.5 (58.3, 80.8), p = 0.012). The final sample included 52 matched patients. Complications occurred significantly less frequently in the elective group compared to the immediate group (16 (50.0%) vs. 3 (10.0%), p < 0.001). Single-factor analysis of variance showed that complications in hospitalized patients were significantly associated with immediate TEVAR as the sole independent risk factor (odds ratio: 9.000, 95% confidence interval: 2.266-35.752, p = 0.002).
CONCLUSION
In this propensity-score matched analysis of patients undergoing TEVAR for BTAI, elective TEVAR was significantly associated with a lower risk of complication rates. In this study using propensity-score matching, patients who underwent elective TEVAR for BTAI had lower complication rates than immediate TEVAR.
Humans
;
Retrospective Studies
;
Male
;
Aorta, Thoracic/surgery*
;
Female
;
Endovascular Procedures/methods*
;
Wounds, Nonpenetrating/mortality*
;
Middle Aged
;
Adult
;
Aged
;
Injury Severity Score
;
Elective Surgical Procedures
;
Time Factors
;
Treatment Outcome
;
Endovascular Aneurysm Repair
8.Advances in Microfluidic Chip Based on Loop-mediated Isothermal Amplification Technology for Rapid Detection of Foodborne Pathogens
Yan-Zhu PAN ; Jia-Hua TANG ; Yue-Xiang CHAI ; Lu-Xi SHU ; Jia-Xin XIE ; Yan ZHANG
Chinese Journal of Analytical Chemistry 2025;53(9):1447-1456
Loop-mediated isothermal amplification(LAMP)is a newin vitronucleic acid amplification technique,which has been widely used in rapid detection of pathogenic bacteria,with advantages of high efficiency,simple operation and low cost.Microfluidic chip technique is a kind of miniaturized and integrated analytical technology,which integrates automation and high throughput,and can effectively avoid sample cross-contamination and has the advantages of high sample utilization rate and high cost-effectivenes.LAMP combined with microfluidic chip can detect multiple samples of the same pathogen at the same time or single samples of different pathogens at the same time,providing a new method for the rapid,field detection of pathogens.In this paper,the research progresses of LAMP microfluidic chip and its application in rapid detection of pathogenic bacteria in recent years were reviewed,and the future prospect was discussed.
9.Expert consensus on prognostic evaluation of cochlear implantation in hereditary hearing loss.
Xinyu SHI ; Xianbao CAO ; Renjie CHAI ; Suijun CHEN ; Juan FENG ; Ningyu FENG ; Xia GAO ; Lulu GUO ; Yuhe LIU ; Ling LU ; Lingyun MEI ; Xiaoyun QIAN ; Dongdong REN ; Haibo SHI ; Duoduo TAO ; Qin WANG ; Zhaoyan WANG ; Shuo WANG ; Wei WANG ; Ming XIA ; Hao XIONG ; Baicheng XU ; Kai XU ; Lei XU ; Hua YANG ; Jun YANG ; Pingli YANG ; Wei YUAN ; Dingjun ZHA ; Chunming ZHANG ; Hongzheng ZHANG ; Juan ZHANG ; Tianhong ZHANG ; Wenqi ZUO ; Wenyan LI ; Yongyi YUAN ; Jie ZHANG ; Yu ZHAO ; Fang ZHENG ; Yu SUN
Journal of Clinical Otorhinolaryngology Head and Neck Surgery 2025;39(9):798-808
Hearing loss is the most prevalent disabling disease. Cochlear implantation(CI) serves as the primary intervention for severe to profound hearing loss. This consensus systematically explores the value of genetic diagnosis in the pre-operative assessment and efficacy prognosis for CI. Drawing upon domestic and international research and clinical experience, it proposes an evidence-based medicine three-tiered prognostic classification system(Favorable, Marginal, Poor). The consensus focuses on common hereditary non-syndromic hearing loss(such as that caused by mutations in genes like GJB2, SLC26A4, OTOF, LOXHD1) and syndromic hereditary hearing loss(such as Jervell & Lange-Nielsen syndrome and Waardenburg syndrome), which are closely associated with congenital hearing loss, analyzing the impact of their pathological mechanisms on CI outcomes. The consensus provides recommendations based on multiple round of expert discussion and voting. It emphasizes that genetic diagnosis can optimize patient selection, predict prognosis, guide post-operative rehabilitation, offer stratified management strategies for patients with different genotypes, and advance the application of precision medicine in the field of CI.
Humans
;
Cochlear Implantation
;
Prognosis
;
Hearing Loss/surgery*
;
Consensus
;
Connexin 26
;
Mutation
;
Sulfate Transporters
;
Connexins/genetics*
10.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
;
Leukodystrophy, Metachromatic/genetics*
;
Pilot Projects
;
Genetic Therapy/methods*
;
Hematopoietic Stem Cell Transplantation
;
Male
;
Follow-Up Studies
;
Female
;
Lentivirus/genetics*
;
Child
;
Child, Preschool
;
Hematopoietic Stem Cells/metabolism*
;
Cerebroside-Sulfatase/metabolism*
;
Adolescent

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