HK1247641A1 - 变体RNAi - Google Patents

变体RNAi Download PDF

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Publication number
HK1247641A1
HK1247641A1 HK18107116.4A HK18107116A HK1247641A1 HK 1247641 A1 HK1247641 A1 HK 1247641A1 HK 18107116 A HK18107116 A HK 18107116A HK 1247641 A1 HK1247641 A1 HK 1247641A1
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HK
Hong Kong
Prior art keywords
rnai
guide sequence
recombinant
disease
further provided
Prior art date
Application number
HK18107116.4A
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English (en)
Chinese (zh)
Inventor
Lisa M. STANEK
Adam Palermo
Brenda RICHARDS
Sergio Pablo SARDI
Catherine O'riordan
Antonius SONG
Original Assignee
Genzyme Corporation
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Publication of HK1247641A1 publication Critical patent/HK1247641A1/zh

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61P25/00—Drugs for disorders of the nervous system
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    • A—HUMAN NECESSITIES
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P35/00—Antineoplastic agents
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C12N15/09—Recombinant DNA-technology
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
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    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • 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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  • Organic Chemistry (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
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  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
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  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Gastroenterology & Hepatology (AREA)
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HK18107116.4A 2015-02-10 2016-02-09 变体RNAi HK1247641A1 (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10
US62/114,578 2015-02-10
PCT/US2016/017207 WO2016130589A2 (en) 2015-02-10 2016-02-09 VARIANT RNAi

Publications (1)

Publication Number Publication Date
HK1247641A1 true HK1247641A1 (zh) 2018-09-28

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HK18107116.4A HK1247641A1 (zh) 2015-02-10 2016-02-09 变体RNAi

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US (4) US10450563B2 (enExample)
EP (1) EP3256588A2 (enExample)
JP (2) JP2018506304A (enExample)
KR (1) KR102670852B1 (enExample)
CN (1) CN107438671B (enExample)
AR (1) AR103646A1 (enExample)
AU (3) AU2016219396B2 (enExample)
CA (2) CA3255657A1 (enExample)
CL (1) CL2017002027A1 (enExample)
CO (1) CO2017009083A2 (enExample)
CR (1) CR20170406A (enExample)
EA (1) EA201791805A1 (enExample)
EC (1) ECSP17059343A (enExample)
HK (1) HK1247641A1 (enExample)
IL (1) IL253893B (enExample)
MA (1) MA40819B1 (enExample)
MX (2) MX2017010369A (enExample)
MY (2) MY194175A (enExample)
PE (1) PE20171382A1 (enExample)
PH (1) PH12017501432A1 (enExample)
SG (3) SG10201907396XA (enExample)
TN (1) TN2017000354A1 (enExample)
TW (1) TWI781079B (enExample)
UY (1) UY36554A (enExample)
WO (1) WO2016130589A2 (enExample)

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