JP2024529425A - ＣＲＩＳＰＲ／Ｃａｓ編集系のためのガイドＲＮＡ - Google Patents

ＣＲＩＳＰＲ／Ｃａｓ編集系のためのガイドＲＮＡ Download PDF

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JP2024529425A
JP2024529425A JP2024504461A JP2024504461A JP2024529425A JP 2024529425 A JP2024529425 A JP 2024529425A JP 2024504461 A JP2024504461 A JP 2024504461A JP 2024504461 A JP2024504461 A JP 2024504461A JP 2024529425 A JP2024529425 A JP 2024529425A
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grna
adenosine deaminase
nls
composition
amino acid
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JP2024529425A5 (enExample
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キャファティ、ブライアン
パッカー、マイケル
アラティン－シャウス、イヴォンヌ
チェン、ロー－イ
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ビーム セラピューティクス インク．
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    • 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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    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3513—Protein; Peptide
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Obesity (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2024504461A 2021-07-23 2022-07-22 ＣＲＩＳＰＲ／Ｃａｓ編集系のためのガイドＲＮＡ Pending JP2024529425A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163225322P 2021-07-23 2021-07-23
US63/225,322 2021-07-23
US202163255927P 2021-10-14 2021-10-14
US63/255,927 2021-10-14
PCT/US2022/074041 WO2023004409A1 (en) 2021-07-23 2022-07-22 Guide rnas for crispr/cas editing systems

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JP2024529425A true JP2024529425A (ja) 2024-08-06
JP2024529425A5 JP2024529425A5 (enExample) 2025-07-18

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US (1) US20240301405A1 (enExample)
EP (1) EP4373931A1 (enExample)
JP (1) JP2024529425A (enExample)
KR (1) KR20240037299A (enExample)
AU (1) AU2022313315A1 (enExample)
CA (1) CA3226664A1 (enExample)
WO (1) WO2023004409A1 (enExample)

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Publication number Priority date Publication date Assignee Title
US20230383277A1 (en) * 2020-10-14 2023-11-30 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
WO2024220980A2 (en) * 2023-04-20 2024-10-24 The Schepens Eye Research Institute, Inc. Dscam inhibitory nucleic acids and methods of use thereof
JP2026523341A (ja) 2023-05-15 2026-07-14 エヌクロマ・バイオ，インコーポレーテッド Ｈｂｖ遺伝子発現のエピジェネティック調節のための組成物および方法
CN122161928A (zh) * 2023-10-25 2026-06-05 上海尧唐生物科技股份有限公司 一种脱氨酶、包含其的碱基编辑器及其应用

Citations (2)

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US20180273937A1 (en) * 2015-09-28 2018-09-27 North Carolina State University Methods and compositions for sequence specific antimicrobials
WO2020028254A1 (en) * 2018-07-30 2020-02-06 Sarepta Therapeutics, Inc. Trimeric peptides for antisense delivery

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US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
JPH0825869B2 (ja) 1987-02-09 1996-03-13 株式会社ビタミン研究所 抗腫瘍剤包埋リポソ−ム製剤
US4917951A (en) 1987-07-28 1990-04-17 Micro-Pak, Inc. Lipid vesicles formed of surfactants and steroids
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US20240301405A1 (en) 2024-09-12
KR20240037299A (ko) 2024-03-21
AU2022313315A1 (en) 2024-02-08
CA3226664A1 (en) 2023-01-26
EP4373931A1 (en) 2024-05-29
WO2023004409A1 (en) 2023-01-26

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