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MA40819B1 - Variant d'arni - Google Patents

Variant d'arni

Info

Publication number
MA40819B1
MA40819B1 MA40819A MA40819A MA40819B1 MA 40819 B1 MA40819 B1 MA 40819B1 MA 40819 A MA40819 A MA 40819A MA 40819 A MA40819 A MA 40819A MA 40819 B1 MA40819 B1 MA 40819B1
Authority
MA
Morocco
Prior art keywords
sequence
arni
guide
relates
opposite
Prior art date
Application number
MA40819A
Other languages
English (en)
Other versions
MA40819A1 (fr
Inventor
Catherine O'riordan
Lisa Stanek
Adam Palermo
Brenda Richards
Sergio Sardi
Antonius Song
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of MA40819A1 publication Critical patent/MA40819A1/fr
Publication of MA40819B1 publication Critical patent/MA40819B1/fr

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector

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Abstract

La présente invention concerne des molécules d'arni comprenant un premier brin contenant une séquence de guidage et un second brin comprenant une séquence qui n'est pas une séquence de guidage, la séquence qui n'est pas une séquence de guidage contenant un renflement à l'opposé de la région d'ensemencement des séquences de guidage ; p. Ex., à l'opposé de la séquence de clivage. Selon certains aspects, l'invention concerne un arni destiné à traiter la maladie de huntington. L'invention concerne en outre de
MA40819A 2015-02-10 2016-02-09 Variant d'arni MA40819B1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10
PCT/US2016/017207 WO2016130589A2 (fr) 2015-02-10 2016-02-09 Variant d'arni

Publications (2)

Publication Number Publication Date
MA40819A1 MA40819A1 (fr) 2018-07-31
MA40819B1 true MA40819B1 (fr) 2020-04-30

Family

ID=56614905

Family Applications (1)

Application Number Title Priority Date Filing Date
MA40819A MA40819B1 (fr) 2015-02-10 2016-02-09 Variant d'arni

Country Status (25)

Country Link
US (4) US10450563B2 (fr)
EP (1) EP3256588A2 (fr)
JP (2) JP2018506304A (fr)
KR (1) KR102670852B1 (fr)
CN (1) CN107438671B (fr)
AR (1) AR103646A1 (fr)
AU (3) AU2016219396B2 (fr)
CA (1) CA3255657A1 (fr)
CL (1) CL2017002027A1 (fr)
CO (1) CO2017009083A2 (fr)
CR (1) CR20170406A (fr)
EA (1) EA201791805A1 (fr)
EC (1) ECSP17059343A (fr)
HK (1) HK1247641A1 (fr)
IL (1) IL253893B (fr)
MA (1) MA40819B1 (fr)
MX (2) MX2017010369A (fr)
MY (2) MY181458A (fr)
PE (1) PE20171382A1 (fr)
PH (1) PH12017501432A1 (fr)
SG (3) SG10201907396XA (fr)
TN (1) TN2017000354A1 (fr)
TW (1) TWI781079B (fr)
UY (1) UY36554A (fr)
WO (1) WO2016130589A2 (fr)

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