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231223s2008 xx ||||| 00| ||eng c |
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|a pubmed25n0622.xml
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|a (NLM)19238792
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|a DE-627
|b ger
|c DE-627
|e rakwb
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| 041 |
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|a eng
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| 100 |
1 |
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|a Morcos, Paul A
|e verfasserin
|4 aut
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| 245 |
1 |
0 |
|a Vivo-Morpholinos
|b a non-peptide transporter delivers Morpholinos into a wide array of mouse tissues
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| 264 |
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1 |
|c 2008
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| 336 |
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|a Text
|b txt
|2 rdacontent
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| 337 |
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|a ohne Hilfsmittel zu benutzen
|b n
|2 rdamedia
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| 338 |
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|a Band
|b nc
|2 rdacarrier
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| 500 |
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|a Date Completed 17.03.2009
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| 500 |
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|a Date Revised 07.06.2018
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| 500 |
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|a published: Print
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| 500 |
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|a Citation Status MEDLINE
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| 520 |
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|a We have developed a new transporter structure that provides effective delivery of Morpholino antisense oligomers into a wide variety of tissues in living mice. This transporter comprises a dendritic structure assembled around a triazine core which serves to position eight guanidinium head groups in a conformation effective to penetrate cell membranes. This transporter structure is conjugated to a Morpholino oligomer to form a delivery-enabled product referred to as a Vivo-Morpholino. Vivo-Morpholinos are shown to effectively enter and function within cultured cells in the presence of 100% serum using a rigorous positive test system based on correction of a defined splicing error in a pre-messenger RNA. In addition, Vivo-Morpholinos are demonstrated to enter into a wide variety of tissues in a similar positive test system in transgenic mice, as evidenced by correction of the targeted splicing error in all tissues assessed, including near-complete splice correction in the small intestine, colon, stomach, liver kidney, and a number of muscles. Finally, Vivo-Morpholinos, which target the exon-skipping of exon 23 harboring a premature termination codon in the mdx mouse model, effectively restore the reading frame of dystrophin and restore expression of a functional dystrophin protein
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| 650 |
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4 |
|a Journal Article
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| 650 |
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7 |
|a Dystrophin
|2 NLM
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| 650 |
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7 |
|a Endo-Porter
|2 NLM
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| 650 |
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7 |
|a Oligonucleotides, Antisense
|2 NLM
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| 650 |
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7 |
|a Peptides
|2 NLM
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| 650 |
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7 |
|a Triazines
|2 NLM
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| 650 |
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7 |
|a Green Fluorescent Proteins
|2 NLM
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| 650 |
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7 |
|a 147336-22-9
|2 NLM
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| 650 |
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7 |
|a Guanidine
|2 NLM
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| 650 |
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7 |
|a JU58VJ6Y3B
|2 NLM
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| 700 |
1 |
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|a Li, Yongfu
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Jiang, Shan
|e verfasserin
|4 aut
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| 773 |
0 |
8 |
|i Enthalten in
|t BioTechniques
|d 1991
|g 45(2008), 6 vom: 25. Dez., Seite 613-4, 616, 618 passim
|w (DE-627)NLM012627046
|x 1940-9818
|7 nnas
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| 773 |
1 |
8 |
|g volume:45
|g year:2008
|g number:6
|g day:25
|g month:12
|g pages:613-4, 616, 618 passim
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|d 45
|j 2008
|e 6
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|h 613-4, 616, 618 passim
|