RU2690333C2 - Соединения и способы модуляции экспрессии дистрофической миотонин-протеинкиназы (dmpk) - Google Patents
Соединения и способы модуляции экспрессии дистрофической миотонин-протеинкиназы (dmpk) Download PDFInfo
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| CN108779466B (zh) | 2015-11-30 | 2024-03-29 | 杜克大学 | 用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法 |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
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| US11427838B2 (en) | 2016-06-29 | 2022-08-30 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders |
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| WO2018017754A1 (en) | 2016-07-19 | 2018-01-25 | Duke University | Therapeutic applications of cpf1-based genome editing |
| CN110177544A (zh) | 2016-11-29 | 2019-08-27 | 普尔泰克健康有限公司 | 用于递送治疗剂的外泌体 |
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| EP3599844A4 (en) * | 2017-06-07 | 2021-01-13 | University of Massachusetts | ANTI-ADAM33 OLIGONUCLEOTIDES AND RELATED PROCESSES |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| EP3808849A1 (en) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| MA51103A (fr) | 2017-12-06 | 2020-10-14 | Avidity Biosciences Inc | Compositions et procédés de traitement de l'atrophie musculaire et de la dystrophie myotonique |
| US12509492B2 (en) | 2018-01-19 | 2025-12-30 | Duke University | Genome engineering with CRISPR-Cas systems in eukaryotes |
| KR20250099758A (ko) | 2018-01-31 | 2025-07-02 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 2c형 지대근이영양증에 대한 유전자 치료 |
| AU2019218987B2 (en) | 2018-02-12 | 2025-04-24 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| KR20210028162A (ko) | 2018-06-29 | 2021-03-11 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 |
| US12370264B1 (en) | 2018-08-02 | 2025-07-29 | Dyne Therapeutics, Inc. | Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| IL321905A (en) * | 2018-08-02 | 2025-09-01 | Dyne Therapeutics Inc | Muscle-targeted conjugates and their uses in the treatment of myotonia dystrophica |
| US12492402B2 (en) | 2018-08-22 | 2025-12-09 | Research Institute At Nationwide Children's Hospital | Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3′ untranslated region of the DMPK gene |
| IL319265A (en) | 2018-12-21 | 2025-04-01 | Avidity Biosciences Inc | Anti-transferrin receptor antibodies and their uses |
| AU2020229340A1 (en) | 2019-02-26 | 2021-09-16 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy |
| US12497615B2 (en) | 2019-04-25 | 2025-12-16 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| EP4017871B1 (en) | 2019-08-21 | 2024-01-17 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| IL310900A (en) | 2020-03-19 | 2024-04-01 | Avidity Biosciences Inc | Preparations and methods for the treatment of facial, back and arm muscle atrophy |
| TW202202173A (zh) | 2020-03-27 | 2022-01-16 | 美商亞維代堤生物科學公司 | 治療肌肉萎縮症之組合物及方法 |
| JP2023130528A (ja) * | 2020-06-24 | 2023-09-21 | 田辺三菱製薬株式会社 | Ｐｌｐ１発現を調節するための化合物、方法及び医薬組成物 |
| US20240117356A1 (en) | 2020-12-31 | 2024-04-11 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11969475B2 (en) * | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11633498B2 (en) * | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| EP4396351A4 (en) * | 2021-09-01 | 2025-12-24 | Ionis Pharmaceuticals Inc | COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION |
| US11833221B2 (en) | 2021-09-01 | 2023-12-05 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds for reducing DMPK expression |
| WO2023043953A1 (en) | 2021-09-16 | 2023-03-23 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| US12285497B2 (en) | 2021-10-15 | 2025-04-29 | Research Institute At Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
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