TW201839136A - 治療血色素異常症之組合物及方法 - Google Patents

治療血色素異常症之組合物及方法 Download PDF

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TW201839136A
TW201839136A TW107103865A TW107103865A TW201839136A TW 201839136 A TW201839136 A TW 201839136A TW 107103865 A TW107103865 A TW 107103865A TW 107103865 A TW107103865 A TW 107103865A TW 201839136 A TW201839136 A TW 201839136A
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cell
seq
cells
grna
molecule
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克雷格 史蒂芬 麥肯寧
克里斯頓 史密特
珍妮佛 斯尼德
蘇珊C 史蒂芬森
毅 楊
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瑞士商諾華公司
美商英特利亞醫療公司
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    • C12N15/09—Recombinant DNA-technology
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    • 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
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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
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    • C12N15/102—Mutagenizing nucleic acids
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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TW107103865A 2017-02-06 2018-02-02 治療血色素異常症之組合物及方法 TW201839136A (zh)

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US201762455464P 2017-02-06 2017-02-06
US62/455,464 2017-02-06

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EP (1) EP3577223A1 (enExample)
JP (3) JP2020505934A (enExample)
KR (2) KR20240017098A (enExample)
CN (2) CN119286853A (enExample)
AR (1) AR110962A1 (enExample)
AU (3) AU2018215726B2 (enExample)
CA (1) CA3052275A1 (enExample)
EA (1) EA201991862A1 (enExample)
IL (2) IL268406B2 (enExample)
MX (2) MX2019009361A (enExample)
MY (1) MY203407A (enExample)
PH (1) PH12019501812A1 (enExample)
SA (1) SA519402400B1 (enExample)
SG (1) SG11201907056XA (enExample)
TW (1) TW201839136A (enExample)
WO (1) WO2018142364A1 (enExample)

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