RNAi COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH'S ATAXIA

The invention provides ribonucleic acid (RNA) molecules, such as siRNA and shRNA molecules, that compensate for a frataxin deficiency or mutation, expression vectors encoding the same, and methods of using the same (e.g., to treat Friedreich's ataxia). L'invention concerne des molécules d&...

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Bibliographic Details
Main Authors WILSON, ROBERT B, XIA, SHUJUAN, COTTICELLI, MARIA GRAZIA, ACQUAVIVA, FABIO, WANG, YONGPING
Format Patent
LanguageEnglish
French
Published 05.11.2015
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Summary:The invention provides ribonucleic acid (RNA) molecules, such as siRNA and shRNA molecules, that compensate for a frataxin deficiency or mutation, expression vectors encoding the same, and methods of using the same (e.g., to treat Friedreich's ataxia). L'invention concerne des molécules d'acide ribonucléique (ARN), telles que des molécules d'ARNsi et d'ARNsh, qui compensent le déficit ou la mutation de frataxine, des vecteurs d'expression codant pour celles-ci, et des procédés d'utilisation associés (par exemple pour traiter l'ataxie de Friedreich).
Bibliography:Application Number: WO2014US49677