Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis.
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Cystinosis: a reviewCell-based therapies for experimental chronic kidney disease: a systematic review and meta-analysisIs genetic rescue of cystinosis an achievable treatment goal?Nephropathic cystinosis: an international consensus documentBrief reports: Lysosomal cross-correction by hematopoietic stem cell-derived macrophages via tunneling nanotubesWorldwide view of nephropathic cystinosis: results from a survey from 30 countriesGene therapy for lysosomal storage disorders: a good start.Clinical utility of chitotriosidase enzyme activity in nephropathic cystinosis.That eagle covering me: transitioning and connected autonomy for emerging adults with cystinosis.A mouse model suggests two mechanisms for thyroid alterations in infantile cystinosis: decreased thyroglobulin synthesis due to endoplasmic reticulum stress/unfolded protein response and impaired lysosomal processing.Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell TransplantationHematopoietic Stem Cells Transplantation Can Normalize Thyroid Function in a Cystinosis Mouse ModelLysosome dysfunction in the pathogenesis of kidney diseases.Improving the prognosis of nephropathic cystinosis.Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.The renal Fanconi syndrome in cystinosis: pathogenic insights and therapeutic perspectives.Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich's ataxia.Gyrfalcons Falco rusticolus adjust CTNS expression to food abundance: a possible contribution to cysteine homeostasis.Cystinosin, the small GTPase Rab11, and the Rab7 effector RILP regulate intracellular trafficking of the chaperone-mediated autophagy receptor LAMP2A.Management dilemmas in pediatric nephrology: Cystinosis.Nephropathic Cystinosis: Symptoms, Treatment, and Perspectives of a Systemic Disease.
P2860
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P2860
Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis.
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
2012年论文
@zh
2012年论文
@zh-cn
name
Hematopoietic stem cell gene t ...... l storage disorder cystinosis.
@ast
Hematopoietic stem cell gene t ...... l storage disorder cystinosis.
@en
type
label
Hematopoietic stem cell gene t ...... l storage disorder cystinosis.
@ast
Hematopoietic stem cell gene t ...... l storage disorder cystinosis.
@en
prefLabel
Hematopoietic stem cell gene t ...... l storage disorder cystinosis.
@ast
Hematopoietic stem cell gene t ...... l storage disorder cystinosis.
@en
P2093
P2860
P50
P356
P1433
P1476
Hematopoietic stem cell gene t ...... l storage disorder cystinosis.
@en
P2093
Brian A Yeagy
Daniel R Salomon
Frank Harrison
P2860
P304
P356
10.1038/MT.2012.214
P577
2012-10-23T00:00:00Z