CN115916973A - 用于治疗具有kras突变的癌症的组合物及其用途 - Google Patents

用于治疗具有kras突变的癌症的组合物及其用途 Download PDF

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CN115916973A
CN115916973A CN202180044035.3A CN202180044035A CN115916973A CN 115916973 A CN115916973 A CN 115916973A CN 202180044035 A CN202180044035 A CN 202180044035A CN 115916973 A CN115916973 A CN 115916973A
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peptide
seq
genome editing
cell penetrating
guide rna
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N·P·德赛
G·迪维塔
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Aadigen LLC
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Aadigen LLC
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Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020214846A1 (en) 2019-04-17 2020-10-22 Aadigen, Llc Peptides and nanoparticles for intracellular delivery of molecules
US20250228953A1 (en) * 2021-11-08 2025-07-17 Entrada Therapeutics, Inc. Intracellular targeting of oligonucleotides
WO2023114887A2 (en) * 2021-12-15 2023-06-22 The Trustees Of Indiana University Cell targeting compositions and methods
JP2023176123A (ja) * 2022-05-31 2023-12-13 株式会社 光バイオ ウイルスベクター及びそれを含むがん細胞増殖抑制剤
CN119384498A (zh) * 2022-06-16 2025-01-28 因特利亚治疗公司 用于对细胞进行遗传修饰的方法和组合物
WO2024187174A2 (en) 2023-03-09 2024-09-12 Aadigen, Llc Compositions for treating cancer with kras mutations and uses thereof
EP4777526A1 (en) * 2023-09-12 2026-07-22 Aadigen, LLC Methods and compositions for treating or preventing cancer
WO2025193628A2 (en) 2024-03-09 2025-09-18 Aadigen, Llc Compositions for treating cancer with kras mutations and uses thereof
CN120005043B (zh) * 2025-02-17 2026-07-17 山东第一医科大学附属肿瘤医院(山东省肿瘤防治研究院、山东省肿瘤医院) 一种用于抑制PRDX1 O-GlcNAc修饰的短肽及其应用

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016049024A2 (en) * 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2018009525A1 (en) * 2016-07-05 2018-01-11 The Johnson Hopkins University Crispr/cas9-based compositions and methods for treating cancer
CN109689677A (zh) * 2016-05-27 2019-04-26 阿迪根有限公司 用于基因组编辑分子的细胞内递送的肽和纳米颗粒
US20190255094A1 (en) * 2016-10-14 2019-08-22 Industry-Academic Cooperation Foundation, Yonsei University Guide rna complementary to kras gene, and use thereof
WO2019186275A1 (en) * 2018-03-27 2019-10-03 G+Flas Life Sciences Sequence-specific in vivo cell targeting

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR100666099B1 (ko) * 2004-04-27 2007-01-10 국립암센터 Ｋ―ｒａｓ 올리고뉴클레오티드 마이크로어레이 및 이를이용하여 Ｋ―ｒａｓ 유전자의 돌연변이를 검사하는 방법
US8889642B2 (en) * 2012-04-19 2014-11-18 Silenseed Ltd. Methods and compositions for RNAi-based cancer treatment
KR101796036B1 (ko) * 2016-12-29 2017-11-10 주식회사 무진메디 Cas9 단백질, KRAS 유전자의 발현을 억제하는 가이드 RNA 및 양이온성 폴리머의 복합체가 봉입된 나노 리포좀 전달체 조성물 또는 이를 함유하는 KRAS 유전자 변이에 따른 항암제 저항성 대장암 치료제
JP2020530463A (ja) * 2017-08-10 2020-10-22 アーディジェン， エルエルシー ウイルスの細胞内送達のためのペプチドおよびナノ粒子
JP7395483B2 (ja) * 2017-10-16 2023-12-11 アーディジェン， エルエルシー ｍＲＮＡの細胞内送達のためのペプチドおよびナノ粒子

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016049024A2 (en) * 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
CN109689677A (zh) * 2016-05-27 2019-04-26 阿迪根有限公司 用于基因组编辑分子的细胞内递送的肽和纳米颗粒
WO2018009525A1 (en) * 2016-07-05 2018-01-11 The Johnson Hopkins University Crispr/cas9-based compositions and methods for treating cancer
US20190255094A1 (en) * 2016-10-14 2019-08-22 Industry-Academic Cooperation Foundation, Yonsei University Guide rna complementary to kras gene, and use thereof
WO2019186275A1 (en) * 2018-03-27 2019-10-03 G+Flas Life Sciences Sequence-specific in vivo cell targeting

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
EVA LENTSCH ET AL.: "CRISPR/Cas9-Mediated Knock-Out of KrasG12D Mutated Pancreatic Cancer Cell Lines", INT J MOL SCI ., vol. 20, no. 22, 14 November 2019 (2019-11-14), pages 1 - 11 *
WOOKJAE LEE ET AL.: "Selective targeting of KRAS oncogenic alleles by CRISPR/Cas9 inhibits proliferation of cancer cells", SCI REP ., vol. 8, no. 1, 8 August 2018 (2018-08-08), pages 1 - 7 *

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