JP7631215B2 - Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 - Google Patents

Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 Download PDF

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JP7631215B2
JP7631215B2 JP2021557397A JP2021557397A JP7631215B2 JP 7631215 B2 JP7631215 B2 JP 7631215B2 JP 2021557397 A JP2021557397 A JP 2021557397A JP 2021557397 A JP2021557397 A JP 2021557397A JP 7631215 B2 JP7631215 B2 JP 7631215B2
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lipid
orf
rna
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JP2022525428A (ja
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チャン，ヨン
シー． アレクサンダー，セス
エム． ウッド，クリスティ
マヘンドラ プラカーシュ カンジョリア，アルティ
オダテ，ショブ
リン サイツァー，ジェシカ
マイケル レスカルボー，レイナード
ストラップス，ワルター
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インテリア セラピューティクス，インコーポレイテッド
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JP2021557397A 2019-03-28 2020-03-27 Ｔｔｒガイドｒｎａと、ｒｎａガイドｄｎａ結合剤をコードするポリヌクレオチドとを含む組成物および方法 Active JP7631215B2 (ja)

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US201962825637P 2019-03-28 2019-03-28
US62/825,637 2019-03-28
PCT/US2020/025513 WO2020198697A1 (en) 2019-03-28 2020-03-27 Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent

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WO2022271780A1 (en) * 2021-06-22 2022-12-29 Intellia Therapeutics, Inc. Methods for in vivo editing of a liver gene
CN118974256A (zh) 2022-03-29 2024-11-15 锐正基因(苏州)有限公司 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法
WO2023230587A2 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
WO2024003805A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Methods and compositions for ttr gene editing and therapy using crispr system
EP4590820A2 (en) 2022-09-22 2025-07-30 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease
EP4627083A1 (en) * 2022-11-30 2025-10-08 Arbor Biotechnologies, Inc. Reverse transcriptase-mediated genetic editing of transthyretin (ttr) and uses thereof
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
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