Muscle and heart function restoration in a limb girdle muscular dystrophy 2I (LGMD2I) mouse model by systemic FKRP gene delivery.
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Efficacy of Gene Therapy Is Dependent on Disease Progression in Dystrophic Mice with Mutations in the FKRP GeneProgress and prospects of gene therapy clinical trials for the muscular dystrophies.Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy.Postnatal Gene Therapy Improves Spatial Learning Despite the Presence of Neuronal Ectopia in a Model of Neuronal Migration Disorder.AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression.B4GALNT2 (GALGT2) Gene Therapy Reduces Skeletal Muscle Pathology in the FKRP P448L Mouse Model of Limb Girdle Muscular Dystrophy 2I.Sexually dimorphic skeletal muscle and cardiac dysfunction in a mouse model of limb girdle muscular dystrophy 2i.Skeletal muscle contractile properties in a novel murine model for limb girdle muscular dystrophy 2i.Autologous intramuscular transplantation of engineered satellite cells induces exosome-mediated systemic expression of Fukutin-related protein and rescues disease phenotype in a murine model of limb-girdle muscular dystrophy type 2I.Skeletal, cardiac, and respiratory muscle function and histopathology in the P448Lneo- mouse model of FKRP-deficient muscular dystrophy.Amelioration of Muscle and Nerve Pathology in LAMA2 Muscular Dystrophy by AAV9-Mini-Agrin.Overexpression of Mutant FKRP Restores Functional Glycosylation and Improves Dystrophic Phenotype in FKRP Mutant Mice.Whole-exome sequencing identifies novel pathogenic mutations and putative phenotype-influencing variants in Polish limb-girdle muscular dystrophy patients.Ribitol restores functionally glycosylated α-dystroglycan and improves muscle function in dystrophic FKRP-mutant miceDose-Dependent Effects of FKRP Gene-Replacement Therapy on Functional Rescue and Longevity in Dystrophic MiceA limb-girdle muscular dystrophy 2I model of muscular dystrophy identifies corrective drug compounds for dystroglycanopathies
P2860
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P2860
Muscle and heart function restoration in a limb girdle muscular dystrophy 2I (LGMD2I) mouse model by systemic FKRP gene delivery.
description
2014 nî lūn-bûn
@nan
2014年の論文
@ja
2014年論文
@yue
2014年論文
@zh-hant
2014年論文
@zh-hk
2014年論文
@zh-mo
2014年論文
@zh-tw
2014年论文
@wuu
2014年论文
@zh
2014年论文
@zh-cn
name
Muscle and heart function rest ...... y systemic FKRP gene delivery.
@en
type
label
Muscle and heart function rest ...... y systemic FKRP gene delivery.
@en
prefLabel
Muscle and heart function rest ...... y systemic FKRP gene delivery.
@en
P2093
P2860
P356
P1433
P1476
Muscle and heart function rest ...... y systemic FKRP gene delivery.
@en
P2093
Carrie Bette Martin
Chi-Hsien Wang
Chunping Qiao
Chunxia Zhao
Hiroyuki Awano
Jianbin Li
Peijuan Lu
P2860
P304
P356
10.1038/MT.2014.141
P577
2014-07-22T00:00:00Z