Oligómero antisentido que induce la omisión del exón 51 en el gen de distrofina humana

La presente memoria descriptiva proporciona un fármaco que provoca una omisión altamente eficaz del exón 51 en el gen de la distrofina humana. La presente memoria descriptiva proporciona un oligómero antisentido que tiene una actividad para inducir la omisión del exón 51 en el gen de la distrofina h...

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Hauptverfasser: Takahiro FUKUI, Yoshitsugu AOKI, Yu HONDA, Kaname MUCHIMA, Shin'ichi TAKEDA, Saki HASEGAWA
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creator Takahiro FUKUI
Yoshitsugu AOKI
Yu HONDA
Kaname MUCHIMA
Shin'ichi TAKEDA
Saki HASEGAWA
description La presente memoria descriptiva proporciona un fármaco que provoca una omisión altamente eficaz del exón 51 en el gen de la distrofina humana. La presente memoria descriptiva proporciona un oligómero antisentido que tiene una actividad para inducir la omisión del exón 51 en el gen de la distrofina humana. The present specification provides a drug that causes highly-efficient skipping of exon 51 in the human dystrophin gene. The present specification provides an antisense oligomer having an activity to induce skipping of exon 51 in the human dystrophin gene.
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
ORGANIC CHEMISTRY
PEPTIDES
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
title Oligómero antisentido que induce la omisión del exón 51 en el gen de distrofina humana
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