Spinal muscular atrophy(SMA),a neurodegenerative disorder caused by mutations in survival motor neuron 1(SMN1)resulting in reduced expression of the survival motor neuron(SMN)protein,is inherited in an autosomal reces...Spinal muscular atrophy(SMA),a neurodegenerative disorder caused by mutations in survival motor neuron 1(SMN1)resulting in reduced expression of the survival motor neuron(SMN)protein,is inherited in an autosomal recessive manner.SMA type 1 is the most frequent subtype of SMA characterized by onset before 6 months of life,inability to achieve independent sitting,and rapidly progressive respiratory and bulbar deterioration,causing a mortality of>90%before 2 years of age.[1]Currently,there have been two lifelong splice-modifying therapies(nusinersen and risdiplam),and a single-dose gene replacement therapy via intravenous onasemnogene abeparvovec for SMA type 1 patients.展开更多
Trillions of microbes reside in the human body and participate in multiple physiological and pathophysiological processes that affect host health throughout the life cycle. The microbiome is hallmarked by distinctive ...Trillions of microbes reside in the human body and participate in multiple physiological and pathophysiological processes that affect host health throughout the life cycle. The microbiome is hallmarked by distinctive compositional and functional features across different life periods.Accumulating evidence has shown that microbes residing in the human body may play fundamental roles in infant development and the maturation of the immune system. Gut microbes are thought to be essential for the facilitation of infantile and childhood development and immunity by assisting in breaking down food substances to liberate nutrients, protecting against pathogens, stimulating or modulating the immune system, and exerting control over the hypothalamic–pituitary–adrenal axis.This review aims to summarize the current understanding of the colonization and development of the gut microbiota in early life, highlighting the recent findings regarding the role of intestinal microbes in pediatric diseases. Furthermore, we also discuss the microbiota-mediated therapeutics that can reconfigure bacterial communities to treat dysbiosis.展开更多
基金supported by the E-Town Cooperation&Development Foundation(No.YCXJ-JZ-2022-007).
文摘Spinal muscular atrophy(SMA),a neurodegenerative disorder caused by mutations in survival motor neuron 1(SMN1)resulting in reduced expression of the survival motor neuron(SMN)protein,is inherited in an autosomal recessive manner.SMA type 1 is the most frequent subtype of SMA characterized by onset before 6 months of life,inability to achieve independent sitting,and rapidly progressive respiratory and bulbar deterioration,causing a mortality of>90%before 2 years of age.[1]Currently,there have been two lifelong splice-modifying therapies(nusinersen and risdiplam),and a single-dose gene replacement therapy via intravenous onasemnogene abeparvovec for SMA type 1 patients.
基金supported by the National Natural Science Foundation of China (Grant Nos. 81671504 and 81401248)the Sanming Project of Medicine in Shenzhen, China (Grant No. SZSM201606088)
文摘Trillions of microbes reside in the human body and participate in multiple physiological and pathophysiological processes that affect host health throughout the life cycle. The microbiome is hallmarked by distinctive compositional and functional features across different life periods.Accumulating evidence has shown that microbes residing in the human body may play fundamental roles in infant development and the maturation of the immune system. Gut microbes are thought to be essential for the facilitation of infantile and childhood development and immunity by assisting in breaking down food substances to liberate nutrients, protecting against pathogens, stimulating or modulating the immune system, and exerting control over the hypothalamic–pituitary–adrenal axis.This review aims to summarize the current understanding of the colonization and development of the gut microbiota in early life, highlighting the recent findings regarding the role of intestinal microbes in pediatric diseases. Furthermore, we also discuss the microbiota-mediated therapeutics that can reconfigure bacterial communities to treat dysbiosis.
