Exon skipping therapy for Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients exp...
Ausführliche Beschreibung
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Kole, Ryszard [verfasserIn] |
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2015transfer abstract |
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4 |
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Enthalten in: Parents’ ambitions and children’s competitiveness - Khadjavi, Menusch ELSEVIER, 2018, Amsterdam [u.a.] |
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volume:87 ; year:2015 ; day:29 ; month:06 ; pages:104-107 ; extent:4 |
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10.1016/j.addr.2015.05.008 |
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| 520 | |a Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. | ||
| 520 | |a Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. | ||
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10.1016/j.addr.2015.05.008 doi GBVA2015006000021.pica (DE-627)ELV023397551 (ELSEVIER)S0169-409X(15)00101-5 DE-627 ger DE-627 rakwb eng 610 610 DE-600 150 300 330 VZ 77.00 bkl Kole, Ryszard verfasserin aut Exon skipping therapy for Duchenne muscular dystrophy 2015transfer abstract 4 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. Krieg, Arthur M. oth Enthalten in Elsevier Science Khadjavi, Menusch ELSEVIER Parents’ ambitions and children’s competitiveness 2018 Amsterdam [u.a.] (DE-627)ELV000099058 volume:87 year:2015 day:29 month:06 pages:104-107 extent:4 https://doi.org/10.1016/j.addr.2015.05.008 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U 77.00 Psychologie: Allgemeines VZ AR 87 2015 29 0629 104-107 4 045F 610 |
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10.1016/j.addr.2015.05.008 doi GBVA2015006000021.pica (DE-627)ELV023397551 (ELSEVIER)S0169-409X(15)00101-5 DE-627 ger DE-627 rakwb eng 610 610 DE-600 150 300 330 VZ 77.00 bkl Kole, Ryszard verfasserin aut Exon skipping therapy for Duchenne muscular dystrophy 2015transfer abstract 4 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. Krieg, Arthur M. oth Enthalten in Elsevier Science Khadjavi, Menusch ELSEVIER Parents’ ambitions and children’s competitiveness 2018 Amsterdam [u.a.] (DE-627)ELV000099058 volume:87 year:2015 day:29 month:06 pages:104-107 extent:4 https://doi.org/10.1016/j.addr.2015.05.008 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U 77.00 Psychologie: Allgemeines VZ AR 87 2015 29 0629 104-107 4 045F 610 |
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10.1016/j.addr.2015.05.008 doi GBVA2015006000021.pica (DE-627)ELV023397551 (ELSEVIER)S0169-409X(15)00101-5 DE-627 ger DE-627 rakwb eng 610 610 DE-600 150 300 330 VZ 77.00 bkl Kole, Ryszard verfasserin aut Exon skipping therapy for Duchenne muscular dystrophy 2015transfer abstract 4 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. Krieg, Arthur M. oth Enthalten in Elsevier Science Khadjavi, Menusch ELSEVIER Parents’ ambitions and children’s competitiveness 2018 Amsterdam [u.a.] (DE-627)ELV000099058 volume:87 year:2015 day:29 month:06 pages:104-107 extent:4 https://doi.org/10.1016/j.addr.2015.05.008 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U 77.00 Psychologie: Allgemeines VZ AR 87 2015 29 0629 104-107 4 045F 610 |
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10.1016/j.addr.2015.05.008 doi GBVA2015006000021.pica (DE-627)ELV023397551 (ELSEVIER)S0169-409X(15)00101-5 DE-627 ger DE-627 rakwb eng 610 610 DE-600 150 300 330 VZ 77.00 bkl Kole, Ryszard verfasserin aut Exon skipping therapy for Duchenne muscular dystrophy 2015transfer abstract 4 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. Krieg, Arthur M. oth Enthalten in Elsevier Science Khadjavi, Menusch ELSEVIER Parents’ ambitions and children’s competitiveness 2018 Amsterdam [u.a.] (DE-627)ELV000099058 volume:87 year:2015 day:29 month:06 pages:104-107 extent:4 https://doi.org/10.1016/j.addr.2015.05.008 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U 77.00 Psychologie: Allgemeines VZ AR 87 2015 29 0629 104-107 4 045F 610 |
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10.1016/j.addr.2015.05.008 doi GBVA2015006000021.pica (DE-627)ELV023397551 (ELSEVIER)S0169-409X(15)00101-5 DE-627 ger DE-627 rakwb eng 610 610 DE-600 150 300 330 VZ 77.00 bkl Kole, Ryszard verfasserin aut Exon skipping therapy for Duchenne muscular dystrophy 2015transfer abstract 4 nicht spezifiziert zzz rdacontent nicht spezifiziert z rdamedia nicht spezifiziert zu rdacarrier Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. Krieg, Arthur M. oth Enthalten in Elsevier Science Khadjavi, Menusch ELSEVIER Parents’ ambitions and children’s competitiveness 2018 Amsterdam [u.a.] (DE-627)ELV000099058 volume:87 year:2015 day:29 month:06 pages:104-107 extent:4 https://doi.org/10.1016/j.addr.2015.05.008 Volltext GBV_USEFLAG_U GBV_ELV SYSFLAG_U 77.00 Psychologie: Allgemeines VZ AR 87 2015 29 0629 104-107 4 045F 610 |
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Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. |
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Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. |
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Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. |
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<?xml version="1.0" encoding="UTF-8"?><collection xmlns="http://www.loc.gov/MARC21/slim"><record><leader>01000caa a22002652 4500</leader><controlfield tag="001">ELV023397551</controlfield><controlfield tag="003">DE-627</controlfield><controlfield tag="005">20230625141228.0</controlfield><controlfield tag="007">cr uuu---uuuuu</controlfield><controlfield tag="008">180603s2015 xx |||||o 00| ||eng c</controlfield><datafield tag="024" ind1="7" ind2=" "><subfield code="a">10.1016/j.addr.2015.05.008</subfield><subfield code="2">doi</subfield></datafield><datafield tag="028" ind1="5" ind2="2"><subfield code="a">GBVA2015006000021.pica</subfield></datafield><datafield tag="035" ind1=" " ind2=" "><subfield code="a">(DE-627)ELV023397551</subfield></datafield><datafield tag="035" ind1=" " ind2=" "><subfield code="a">(ELSEVIER)S0169-409X(15)00101-5</subfield></datafield><datafield tag="040" ind1=" " ind2=" "><subfield code="a">DE-627</subfield><subfield code="b">ger</subfield><subfield code="c">DE-627</subfield><subfield code="e">rakwb</subfield></datafield><datafield tag="041" ind1=" " ind2=" "><subfield code="a">eng</subfield></datafield><datafield tag="082" ind1="0" ind2=" "><subfield code="a">610</subfield></datafield><datafield tag="082" ind1="0" ind2="4"><subfield code="a">610</subfield><subfield code="q">DE-600</subfield></datafield><datafield tag="082" ind1="0" ind2="4"><subfield code="a">150</subfield><subfield code="a">300</subfield><subfield code="a">330</subfield><subfield code="q">VZ</subfield></datafield><datafield tag="084" ind1=" " ind2=" "><subfield code="a">77.00</subfield><subfield code="2">bkl</subfield></datafield><datafield tag="100" ind1="1" ind2=" "><subfield code="a">Kole, Ryszard</subfield><subfield code="e">verfasserin</subfield><subfield code="4">aut</subfield></datafield><datafield tag="245" ind1="1" ind2="0"><subfield code="a">Exon skipping therapy for Duchenne muscular dystrophy</subfield></datafield><datafield tag="264" ind1=" " ind2="1"><subfield code="c">2015transfer abstract</subfield></datafield><datafield tag="300" ind1=" " ind2=" "><subfield code="a">4</subfield></datafield><datafield tag="336" ind1=" " ind2=" "><subfield code="a">nicht spezifiziert</subfield><subfield code="b">zzz</subfield><subfield code="2">rdacontent</subfield></datafield><datafield tag="337" ind1=" " ind2=" "><subfield code="a">nicht spezifiziert</subfield><subfield code="b">z</subfield><subfield code="2">rdamedia</subfield></datafield><datafield tag="338" ind1=" " ind2=" "><subfield code="a">nicht spezifiziert</subfield><subfield code="b">zu</subfield><subfield code="2">rdacarrier</subfield></datafield><datafield tag="520" ind1=" " ind2=" "><subfield code="a">Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. 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