Gene therapy approaches for lysosomal storage disease: next-generation treatment.
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Moving towards effective therapeutic strategies for Neuronal Ceroid LipofuscinosisTargeted approaches to induce immune tolerance for Pompe disease therapyGene therapy on the moveThe effect of injection speed and serial injection on propidium iodide entry into cultured HeLa and primary neonatal fibroblast cells using lance array nanoinjection.Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage diseaseGene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.Development of Gene Transfer for Induction of Antigen-specific Tolerance.Candidate molecules for chemical chaperone therapy of GM1-gangliosidosis.Amyloidosis, synucleinopathy, and prion encephalopathy in a neuropathic lysosomal storage disease: the CNS-biomarker potential of peripheral blood.Pharmacological chaperone therapy for lysosomal storage diseases.Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Adeno-associated virus-mediated gene therapy for metabolic myopathy.Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.Gene-based therapies of neuromuscular disorders: an update and the pivotal role of patient organizations in their discovery and implementation.Gene therapy for the neurological manifestations in lysosomal storage disorders.Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration.Serum global metabolomics profiling reveals profound metabolic impairments in patients with MPS IIIA and MPS IIIB.Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery.Transcriptome assessment of the Pompe (Gaa-/-) mouse spinal cord indicates widespread neuropathology.Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice.Injection parameters and virus dependent choice of promoters to improve neuron targeting in the nonhuman primate brain.Can manipulation of splicing offer gene therapy possibilities to those with tumour-prone disorders?
P2860
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P2860
Gene therapy approaches for lysosomal storage disease: next-generation treatment.
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
Gene therapy approaches for lysosomal storage disease: next-generation treatment.
@ast
Gene therapy approaches for lysosomal storage disease: next-generation treatment.
@en
type
label
Gene therapy approaches for lysosomal storage disease: next-generation treatment.
@ast
Gene therapy approaches for lysosomal storage disease: next-generation treatment.
@en
prefLabel
Gene therapy approaches for lysosomal storage disease: next-generation treatment.
@ast
Gene therapy approaches for lysosomal storage disease: next-generation treatment.
@en
P2093
P2860
P356
P1433
P1476
Gene therapy approaches for lysosomal storage disease: next-generation treatment.
@en
P2093
Barry J Byrne
Cathryn S Mah
Darin J Falk
Nathalie Clément
P2860
P304
P356
10.1089/HUM.2012.140
P577
2012-08-01T00:00:00Z