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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| Main Authors | , , , , |
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| Format | Patent |
| Language | English French |
| Published |
05.11.2015
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| Online Access | Get full text |
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| Abstract | 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). |
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| AbstractList | 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). |
| Author | WILSON, ROBERT B WANG, YONGPING ACQUAVIVA, FABIO COTTICELLI, MARIA GRAZIA XIA, SHUJUAN |
| Author_xml | – fullname: WILSON, ROBERT B – fullname: XIA, SHUJUAN – fullname: COTTICELLI, MARIA GRAZIA – fullname: ACQUAVIVA, FABIO – fullname: WANG, YONGPING |
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| DocumentTitleAlternate | COMPOSITIONS D'ARNI ET MÉTHODES DE TRAITEMENT DE L'ATAXIE DE FRIEDREICH |
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| Snippet | The invention provides ribonucleic acid (RNA) molecules, such as siRNA and shRNA molecules, that compensate for a frataxin deficiency or mutation, expression... |
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| SubjectTerms | 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 |
| Title | RNAi COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH'S ATAXIA |
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