RNA-based therapeutic approaches for coagulation factor deficiencies.
about
Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.Activation of a cryptic splice site in a potentially lethal coagulation defect accounts for a functional protein variant.Regulation of a strong F9 cryptic 5'ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotidesCharacterization of an apparently synonymous F5 mutation causing aberrant splicing and factor V deficiency.Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based TherapeuticsAn Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants.An engineered U1 small nuclear RNA rescues splicing defective coagulation F7 gene expression in mice.
P2860
Q35796587-685131DE-EECD-48B5-B781-D25CE5DE635DQ37209107-16AF30B5-E0B3-449A-8B6B-ABC5A121350FQ38865127-99F4426A-D59F-4359-A192-62EF46C5F488Q38932615-625F557A-3EE0-4F52-9DF4-B01CFD5B76A7Q39145734-556805B2-0E8B-4792-A39B-76A4E3A47EAEQ41671484-8CCB096F-7E96-4B50-A1D2-1DA3EBE25D7DQ42224211-592E76D3-6211-49D8-BFC5-2E07F72B1D2C
P2860
RNA-based therapeutic approaches for coagulation factor deficiencies.
description
2011 nî lūn-bûn
@nan
2011年の論文
@ja
2011年学术文章
@wuu
2011年学术文章
@zh-cn
2011年学术文章
@zh-hans
2011年学术文章
@zh-my
2011年学术文章
@zh-sg
2011年學術文章
@yue
2011年學術文章
@zh
2011年學術文章
@zh-hant
name
RNA-based therapeutic approaches for coagulation factor deficiencies.
@en
RNA-based therapeutic approaches for coagulation factor deficiencies.
@nl
type
label
RNA-based therapeutic approaches for coagulation factor deficiencies.
@en
RNA-based therapeutic approaches for coagulation factor deficiencies.
@nl
prefLabel
RNA-based therapeutic approaches for coagulation factor deficiencies.
@en
RNA-based therapeutic approaches for coagulation factor deficiencies.
@nl
P2093
P2860
P1476
RNA-based therapeutic approaches for coagulation factor deficiencies.
@en
P2093
P2860
P304
P356
10.1111/J.1538-7836.2011.04481.X
P577
2011-11-01T00:00:00Z