Infectious Delivery and Expression of a 135 kb Human FRDA Genomic DNA Locus Complements Friedreich's Ataxia Deficiency in Human Cells.
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Physiological transgene regulation and functional complementation of a neurological disease gene deficiency in neuronsSmall molecules affecting transcription in Friedreich ataxiaA GAA repeat expansion reporter model of Friedreich's ataxia recapitulates the genomic context and allows rapid screening of therapeutic compounds.Advancements in the pathophysiology of Friedreich's Ataxia and new prospects for treatments.HSV-1 amplicon vectors: a promising and versatile tool for gene delivery.Progress and prospects: biological properties and technological advances of herpes simplex virus type 1-based amplicon vectors.HSV-1-derived amplicon vectors: recent technological improvements and remaining difficulties--a review.Constitutive and Inducible Innate Responses in Cells Infected by HSV-1-Derived Amplicon Vectors.Current and emerging treatment options in the management of Friedreich ataxia.Targeting the central nervous system with herpes simplex virus / Sleeping Beauty hybrid amplicon vectors.Infectious delivery and long-term persistence of transgene expression in the brain by a 135-kb iBAC-FXN genomic DNA expression vector.Therapeutic approaches for the treatment of Friedreich's ataxia.Pharmacological therapeutics in Friedreich ataxia: the present state.Herpes Virus Amplicon Vectors.Conditional gene vectors regulated in cis.Prospects for the use of artificial chromosomes and minichromosome-like episomes in gene therapy.Sustained FXN expression in dorsal root ganglia from a nonreplicative genomic HSV-1 vector.Genomic form of rhodopsin DNA nanoparticles rescued autosomal dominant Retinitis pigmentosa in the P23H knock-in mouse model.FXN methylation predicts expression and clinical outcome in Friedreich ataxia.
P2860
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P2860
Infectious Delivery and Expression of a 135 kb Human FRDA Genomic DNA Locus Complements Friedreich's Ataxia Deficiency in Human Cells.
description
2007 nî lūn-bûn
@nan
2007年の論文
@ja
2007年学术文章
@wuu
2007年学术文章
@zh
2007年学术文章
@zh-cn
2007年学术文章
@zh-hans
2007年学术文章
@zh-my
2007年学术文章
@zh-sg
2007年學術文章
@yue
2007年學術文章
@zh-hant
name
Infectious Delivery and Expres ...... xia Deficiency in Human Cells.
@en
Infectious Delivery and Expres ...... xia Deficiency in Human Cells.
@nl
type
label
Infectious Delivery and Expres ...... xia Deficiency in Human Cells.
@en
Infectious Delivery and Expres ...... xia Deficiency in Human Cells.
@nl
altLabel
Infectious delivery and expres ...... xia deficiency in human cells.
@en
Infectious delivery and expres ...... xia deficiency in human cells.
@nl
prefLabel
Infectious Delivery and Expres ...... xia Deficiency in Human Cells.
@en
Infectious Delivery and Expres ...... xia Deficiency in Human Cells.
@nl
P2093
P356
P1433
P1476
Infectious Delivery and Expres ...... xia Deficiency in Human Cells.
@en
Infectious delivery and expres ...... xia deficiency in human cells.
@en
P2093
Alfredo Gimenez-Cassina
Javier Diaz-Nido
Richard Wade-Martins
Silvia Gomez-Sebastian
P304
P356
10.1038/SJ.MT.6300021
P577
2007-02-01T00:00:00Z