TWI835719B - 經修飾之嚮導rna - Google Patents

經修飾之嚮導rna Download PDF

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TWI835719B
TWI835719B TW106143268A TW106143268A TWI835719B TW I835719 B TWI835719 B TW I835719B TW 106143268 A TW106143268 A TW 106143268A TW 106143268 A TW106143268 A TW 106143268A TW I835719 B TWI835719 B TW I835719B
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nucleotides
region
sgrna
modified
hairpin
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TW106143268A
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TW201825679A (zh
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艾美 麥德生 羅登 史密斯
大衛 V 莫瑞賽
瓦特 史瑞普斯
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美商英特利亞醫療公司
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/30—Chemical structure
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    • C12N2310/322—2'-R Modification
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Pharmacology & Pharmacy (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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TW106143268A 2016-12-08 2017-12-08 經修飾之嚮導rna TWI835719B (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662431756P 2016-12-08 2016-12-08
US62/431,756 2016-12-08

Publications (2)

Publication Number Publication Date
TW201825679A TW201825679A (zh) 2018-07-16
TWI835719B true TWI835719B (zh) 2024-03-21

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US (4) US11479767B2 (enExample)
EP (1) EP3551757A1 (enExample)
JP (4) JP7696694B2 (enExample)
KR (1) KR102595683B1 (enExample)
CN (2) CN119464289A (enExample)
AU (2) AU2017374044B2 (enExample)
BR (1) BR112019011509A2 (enExample)
CA (1) CA3046376A1 (enExample)
CO (1) CO2019007258A2 (enExample)
EA (1) EA201991369A1 (enExample)
IL (1) IL267024B2 (enExample)
MX (2) MX2025000703A (enExample)
MY (1) MY206324A (enExample)
PH (1) PH12019501262A1 (enExample)
SG (1) SG10202106058WA (enExample)
TW (1) TWI835719B (enExample)
WO (1) WO2018107028A1 (enExample)
ZA (1) ZA202007633B (enExample)

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