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Gene therapy for spinal muscular atrophy:perspectives on the possibility of optimizing SMN1 delivery to correct all neurological and systemic perturbations
《Neural Regeneration Research》2025年第7期2011-2012,共2页Sharon J.Brown Rafael J.Yáñez-Muñoz Heidi R.Fuller 
supported by the Faculty Research Fund(Faculty of Medicine&Health Science,Keele University)Career Development Award–(April 2022)(to SJB)。
Spinal muscular atrophy(SMA)is a genetic condition that results in selective lower motor neuron loss with concomitant muscle weakness and atrophy.The genetic cause of SMA was understood in 1995 when loss or impairment...
关键词：ATROPHY traditionally absolute 
Neural differentiation protocols:how to choose the correct approach被引量：1
《Neural Regeneration Research》2023年第6期1273-1274,共2页Michael Telias 
supported by an unrestricted grant to the University of Rochester’s Department of Ophthalmology from the Research to Prevent Blindness Foundation
Pluripotent stem cells in neural differentiation:characterization and potential:The establishment and use of pluripotent stem cells(PSCs),including embryonic(ESCs)and induced(iPSCs),constitutes a major scientific brea...
关键词：eventually BREAKTHROUGH APPROACH 
Induced pluripotent stem cells from Huntington's disease patients:a promising approach to define and correct disease-related alterations被引量：3
《Neural Regeneration Research》2019年第5期769-770,共2页Azra Fatima Ricardo Gutiérrez-Garcia David Vilchez 
supported by the Else Kröner-Fresenius-Stiftung(2015_A118)
Adult somatic cells such as skin or blood cells from either health donors or patients can be reprogrammed into induced pluripotent stem cells(iPSCs).Given their unlimited self-renewal and differentiation capacities,iP...
关键词：ADULT SOMATIC CELLS skin or blood CELLS Huntington’s DISEASE PATIENTS 
The role of the unfolded protein response in myelination被引量：1
《Neural Regeneration Research》2016年第3期394-395,共2页Michelle C.Naughton Jill M.McMahon Una F.FitzGerald 
The production,transport and integration of myelin components into the membrane during development is a highly coordinated and regulated process that relies heavily on the endoplasmic reticulum（ER）,a sub-cellular or...
关键词：endoplasmic translational reticulum prior folding assembly coordinated myelin correct tracts 
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