BACKGROUND Ethylmalonic encephalopathy(EE) is a rare autosomal recessive metabolic disorder caused by impaired mitochondrial sulfur dioxygenase. Due to poor therapeutic effect of current conventional treatments, progr...BACKGROUND Ethylmalonic encephalopathy(EE) is a rare autosomal recessive metabolic disorder caused by impaired mitochondrial sulfur dioxygenase. Due to poor therapeutic effect of current conventional treatments, progressive psychomotor regression and neurological impairment usually contribute to early death in the first decade of life. Liver transplantation(LT) is emerging as a novel therapeutic option for EE;however, worldwide experience is still limited.CASE SUMMARY An 18-mo-old patient with the diagnosis of EE received a living donor liver transplant in our institution, which, to our knowledge, is the first case in AsianPacific countries. During 20 mo of follow-up, the longitudinal metabolic evaluations revealed a wild fluctuation in urinary EMA levels, still far beyond the normal range. Urinary 2-methylsuccinic acid levels gradually restored to normal, whereas the concentrations of urinary isobutyrylglycine and plasma C4-and C5-acylcarnitines fluctuated markedly and still remained above the reference limits. Only mild amelioration of petechiae and ecchymosis was observed, and no dramatic reversion of chronic mucoid diarrhea and orthostatic acrocyanosis occurred. Although brain magnetic resonance imaging suggested a certain improvement in basal ganglia lesions, the patient still presented developmental delay and neurologic disability.CONCLUSION LT may bring about a partial but not complete cure to EE. Given its definite role in defending against the devastating natural progression of EE, LT should still be considered for patients with EE in the absence of other superior therapeutic options. Early establishment of diagnosis and initiation of conventional treatment pre-transplant, timely LT, and continuous administration of metabolismcorrecting medications post-transplant may be helpful in minimizing the neurologic impairment and maximizing the therapeutic value of LT in EE.展开更多
One key of constructing ideal transdermal drug delivery system(TDDS)is enhancing the percutaneous rate of drugs without sacrificing compatibility.Ethosomes(Eths)have excellent transdermal performance as well as good b...One key of constructing ideal transdermal drug delivery system(TDDS)is enhancing the percutaneous rate of drugs without sacrificing compatibility.Ethosomes(Eths)have excellent transdermal performance as well as good biocompatibility,and thus been widely used as drug carrier.Hydrogel has good 3-dimensional mesh structure which is convenience for drugs release and storage.In this study,Eths were introduced into silk fibroin(SF)/polyvinyl alcohol(PVA)composite hydrogel to construct a novel TDDS through a green process.The Ethsomes(Eths)-SF/PVA composite hydrogel TDDS showed good mechanical properties(stress:(0.236±0.032)MPa;strain:(65.74±2.45)%).Also,skin fibroblasts can grow and proliferate well on this TDDS,indicating that this material has a good cytocompatibility.Furthermore,with doxorubicin hydrochloride(Dox)as a model drug loaded in ethosomes,in vitro studies showed that this TDDS was able to transdermally release Dox efficiently.Our data suggested this novel system had a good potential for application in TDD,though further evaluative study still needed to carry out.展开更多
The method to measure the refractive index of the liquid-state metal is introduced. By inserting a wedge sample cell to the optical path of the Michelson interferometer, the refractive index of the liquid-state alloy ...The method to measure the refractive index of the liquid-state metal is introduced. By inserting a wedge sample cell to the optical path of the Michelson interferometer, the refractive index of the liquid-state alloy SCN-Eth is measured at different temperatures and densities. The results show that this method can be applied to measure the refractive index of various liquids, especially at different temperatures.展开更多
基金Supported by Beijing Municipal Science&Technology Commission,No. Z181100001718220Capital's Funds for Health Improvement and Research,No. 2020-1-2024。
文摘BACKGROUND Ethylmalonic encephalopathy(EE) is a rare autosomal recessive metabolic disorder caused by impaired mitochondrial sulfur dioxygenase. Due to poor therapeutic effect of current conventional treatments, progressive psychomotor regression and neurological impairment usually contribute to early death in the first decade of life. Liver transplantation(LT) is emerging as a novel therapeutic option for EE;however, worldwide experience is still limited.CASE SUMMARY An 18-mo-old patient with the diagnosis of EE received a living donor liver transplant in our institution, which, to our knowledge, is the first case in AsianPacific countries. During 20 mo of follow-up, the longitudinal metabolic evaluations revealed a wild fluctuation in urinary EMA levels, still far beyond the normal range. Urinary 2-methylsuccinic acid levels gradually restored to normal, whereas the concentrations of urinary isobutyrylglycine and plasma C4-and C5-acylcarnitines fluctuated markedly and still remained above the reference limits. Only mild amelioration of petechiae and ecchymosis was observed, and no dramatic reversion of chronic mucoid diarrhea and orthostatic acrocyanosis occurred. Although brain magnetic resonance imaging suggested a certain improvement in basal ganglia lesions, the patient still presented developmental delay and neurologic disability.CONCLUSION LT may bring about a partial but not complete cure to EE. Given its definite role in defending against the devastating natural progression of EE, LT should still be considered for patients with EE in the absence of other superior therapeutic options. Early establishment of diagnosis and initiation of conventional treatment pre-transplant, timely LT, and continuous administration of metabolismcorrecting medications post-transplant may be helpful in minimizing the neurologic impairment and maximizing the therapeutic value of LT in EE.
基金Natural Science Foundation of Shanghai,China(No.12ZR1400300)the Innovation Foundation of Donghua University,China(No.EG2015067)+1 种基金the Scientific Research Foundation for the Returned Overseas Chinese Scholars,State Education Ministry,China“111 Project”Biomedical Textile Materials Science and Technology,China(No.B07024)
文摘One key of constructing ideal transdermal drug delivery system(TDDS)is enhancing the percutaneous rate of drugs without sacrificing compatibility.Ethosomes(Eths)have excellent transdermal performance as well as good biocompatibility,and thus been widely used as drug carrier.Hydrogel has good 3-dimensional mesh structure which is convenience for drugs release and storage.In this study,Eths were introduced into silk fibroin(SF)/polyvinyl alcohol(PVA)composite hydrogel to construct a novel TDDS through a green process.The Ethsomes(Eths)-SF/PVA composite hydrogel TDDS showed good mechanical properties(stress:(0.236±0.032)MPa;strain:(65.74±2.45)%).Also,skin fibroblasts can grow and proliferate well on this TDDS,indicating that this material has a good cytocompatibility.Furthermore,with doxorubicin hydrochloride(Dox)as a model drug loaded in ethosomes,in vitro studies showed that this TDDS was able to transdermally release Dox efficiently.Our data suggested this novel system had a good potential for application in TDD,though further evaluative study still needed to carry out.
文摘The method to measure the refractive index of the liquid-state metal is introduced. By inserting a wedge sample cell to the optical path of the Michelson interferometer, the refractive index of the liquid-state alloy SCN-Eth is measured at different temperatures and densities. The results show that this method can be applied to measure the refractive index of various liquids, especially at different temperatures.
