Antisense oligonucleotides rescue aberrant splicing of ABCA4

The present invention relates to the field of medicine. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Stargardt disease.

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Hauptverfasser: Garanto Iglesias, Alejandro, Collin, Robert Wilhelmus Johanna, Cheetham, Michael Edward, Cremers, Franciscus Peter Maria
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creator Garanto Iglesias, Alejandro
Collin, Robert Wilhelmus Johanna
Cheetham, Michael Edward
Cremers, Franciscus Peter Maria
description The present invention relates to the field of medicine. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Stargardt disease.
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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
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
title Antisense oligonucleotides rescue aberrant splicing of ABCA4
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