Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
about
Targeted approaches to induce immune tolerance for Pompe disease therapyAldehyde dehydrogenase 2 in aplastic anemia, Fanconi anemia and hematopoietic stem cellsPompe disease: Shared and unshared features of lysosomal storage disordersCopackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Transcriptome assessment of the Pompe (Gaa-/-) mouse spinal cord indicates widespread neuropathology.A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform.Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice.Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease.13C/31P MRS Metabolic Biomarkers of Disease Progression and Response to AAV Delivery of hGAA in a Mouse Model of Pompe DiseaseSafety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in children affected by Pompe Disease.Locomotor and skeletal muscle abnormalities in trembler J neuropathic mice.Spot light on skeletal muscles: optogenetic stimulation to understand and restore skeletal muscle function.
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P2860
Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
2015年论文
@zh
2015年论文
@zh-cn
name
Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
@ast
Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
@en
type
label
Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
@ast
Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
@en
prefLabel
Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
@ast
Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
@en
P2093
P2860
P356
P1433
P1476
Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
@en
P2093
Adrian G Todd
Barry J Byrne
Darin J Falk
David D Fuller
Glenn A Walter
Jessica A McElroy
Robert W Grange
P2860
P304
P356
10.1002/ANA.24433
P577
2015-06-30T00:00:00Z