CY1121733T1 - Επαγομενη απο rnai καταστολη του γονιδιου της χαντινγκτινης - Google Patents
Επαγομενη απο rnai καταστολη του γονιδιου της χαντινγκτινηςInfo
- Publication number
- CY1121733T1 CY1121733T1 CY20191100616T CY191100616T CY1121733T1 CY 1121733 T1 CY1121733 T1 CY 1121733T1 CY 20191100616 T CY20191100616 T CY 20191100616T CY 191100616 T CY191100616 T CY 191100616T CY 1121733 T1 CY1121733 T1 CY 1121733T1
- Authority
- CY
- Cyprus
- Prior art keywords
- rna
- rnai
- double
- sequence
- huntingtin gene
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title 1
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 4
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 3
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 3
- 230000000295 complement effect Effects 0.000 abstract 2
- 108091030071 RNAI Proteins 0.000 abstract 1
- 238000010171 animal model Methods 0.000 abstract 1
- 230000009368 gene silencing by RNA Effects 0.000 abstract 1
- 230000001939 inductive effect Effects 0.000 abstract 1
- 230000016273 neuron death Effects 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
Η παρούσα εφεύρεση εξασφαλίζει ένα δίκλωνο RNA που περιλαμβάνει μια πρώτη αλληλουχία RNA και μια δεύτερη αλληλουχία RNA όπου η πρώτη και δεύτερη αλληλουχία RNA είναι ουσιαστικά συμπληρωματικές, όπου η πρώτη αλληλουχία RNA έχει ένα μήκος αλληλουχίας τουλάχιστον 19 νουκλεοτίδια και είναι ουσιαστικά συμπληρωματική στην SEQ ID NO. 1. Το ρηθέν δίκλωνο RNA είναι προς χρήση στην επαγωγή RNAi κατά αλληλουχιών του εξονίου 1 της Χαντινγκτίνης. Το δίκλωνο RNA της εφεύρεσης ήταν ικανό μείωσης του νευρωνικού κυτταρικού θανάτου και συσσωματωμάτων της χαντινγκτίνης σε ένα ζωικό μοντέλο.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14200308 | 2014-12-24 | ||
| PCT/EP2015/081157 WO2016102664A1 (en) | 2014-12-24 | 2015-12-23 | Rnai induced huntingtin gene suppression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CY1121733T1 true CY1121733T1 (el) | 2020-07-31 |
Family
ID=52282540
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CY20191100616T CY1121733T1 (el) | 2014-12-24 | 2019-06-13 | Επαγομενη απο rnai καταστολη του γονιδιου της χαντινγκτινης |
Country Status (25)
| Country | Link |
|---|---|
| US (4) | US10174321B2 (el) |
| EP (2) | EP3540063A1 (el) |
| JP (2) | JP6839094B2 (el) |
| KR (1) | KR102636633B1 (el) |
| CN (1) | CN108064292B (el) |
| AU (1) | AU2015370903B2 (el) |
| CA (1) | CA2971920C (el) |
| CO (1) | CO2017007335A2 (el) |
| CY (1) | CY1121733T1 (el) |
| DK (1) | DK3237618T3 (el) |
| EA (1) | EA037696B1 (el) |
| ES (1) | ES2732023T3 (el) |
| HK (1) | HK1246344B (el) |
| HR (1) | HRP20190992T1 (el) |
| HU (1) | HUE043842T2 (el) |
| IL (1) | IL252990B (el) |
| LT (1) | LT3237618T (el) |
| MX (1) | MX381121B (el) |
| PL (1) | PL3237618T3 (el) |
| PT (1) | PT3237618T (el) |
| RS (1) | RS58862B1 (el) |
| SI (1) | SI3237618T1 (el) |
| SM (1) | SMT201900347T1 (el) |
| TR (1) | TR201908969T4 (el) |
| WO (1) | WO2016102664A1 (el) |
Families Citing this family (68)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| JP6128529B2 (ja) | 2011-07-19 | 2017-05-17 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | 官能化核酸の合成のための方法 |
| SG11201500243WA (en) | 2012-07-13 | 2015-04-29 | Shin Nippon Biomedical Lab Ltd | Chiral nucleic acid adjuvant |
| KR101850319B1 (ko) | 2012-07-13 | 2018-04-20 | 웨이브 라이프 사이언시스 리미티드 | 비대칭 보조 그룹 |
| CN112007045A (zh) | 2012-07-13 | 2020-12-01 | 波涛生命科学有限公司 | 手性控制 |
| EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
| EP4137572A1 (en) | 2014-01-16 | 2023-02-22 | Wave Life Sciences Ltd. | Chiral design |
| SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| HK1244299A1 (zh) | 2014-11-14 | 2018-08-03 | Voyager Therapeutics, Inc. | 治療肌萎縮側索硬化(als)的組合物和方法 |
| WO2016102664A1 (en) * | 2014-12-24 | 2016-06-30 | Uniqure Ip B.V. | Rnai induced huntingtin gene suppression |
| US10450563B2 (en) | 2015-02-10 | 2019-10-22 | Genzyme Corporation | Variant RNAi |
| US20160319278A1 (en) | 2015-04-03 | 2016-11-03 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| EA201800367A1 (ru) | 2015-12-10 | 2019-02-28 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Способы лечения болезни хантингтона |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| JP7066635B2 (ja) | 2016-05-18 | 2022-05-13 | ボイジャー セラピューティクス インコーポレイテッド | 調節性ポリヌクレオチド |
| US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| SG11201909868YA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| BR112019025740A2 (pt) | 2017-06-05 | 2020-06-23 | Ptc Therapeutics, Inc. | Compostos para tratar a doença de huntington |
| CA3064590A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | Two-tailed self-delivering sirna and related methods |
| US11382918B2 (en) | 2017-06-28 | 2022-07-12 | Ptc Therapeutics, Inc. | Methods for treating Huntington's Disease |
| MX2019015580A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| MY205041A (en) | 2017-09-22 | 2024-09-29 | Genzyme Corp | Variant rnai |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| CN108384803A (zh) * | 2017-12-19 | 2018-08-10 | 上海百力格生物技术有限公司 | 一种用于miRNA干扰载体构建筛选特异对照的质粒及其构建方法 |
| JP7399870B2 (ja) | 2018-03-27 | 2023-12-18 | ピーティーシー セラピューティクス， インコーポレイテッド | ハンチントン病を処置するための化合物 |
| MA51938B1 (fr) * | 2018-05-15 | 2022-10-31 | Univ Washington | Compositions et procédés pour réduire les anomalies d'épissage et traiter des troubles de dominance arn |
| WO2019222413A1 (en) * | 2018-05-15 | 2019-11-21 | University Of Massachusetts | Modified aav constructs and uses thereof |
| WO2020005873A1 (en) | 2018-06-27 | 2020-01-02 | Ptc Therapeutics, Inc. | Heterocyclic and heteroaryl compounds for treating huntington's disease |
| AU2019294482B2 (en) | 2018-06-27 | 2022-09-01 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating Huntington's disease |
| JP7565218B2 (ja) | 2018-07-02 | 2024-10-10 | ボイジャー セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症および脊髄に関連する障害の治療 |
| JP7627042B2 (ja) | 2018-08-23 | 2025-02-05 | ユニバーシティー オブ マサチューセッツ | Ｏ－メチルリッチ完全安定化オリゴヌクレオチド |
| CA3112007A1 (en) | 2018-09-12 | 2020-03-19 | Uniqure Ip B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
| CA3120177A1 (en) * | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | Method and means to deliver mirna to target cells |
| JP2022507283A (ja) | 2018-11-19 | 2022-01-18 | ユニキュアー アイピー ビー．ブイ． | 脊髄小脳失調症３型の治療のためのアタキシン－３のｒｎａｉ誘導低減 |
| CA3119721A1 (en) | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | A companion diagnostic to monitor the effects of gene therapy |
| CN113302302A (zh) * | 2019-01-09 | 2021-08-24 | 科英布拉大学 | 双链rna及其用途 |
| AU2020207935A1 (en) | 2019-01-18 | 2021-08-26 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| US20220251567A1 (en) | 2019-07-10 | 2022-08-11 | Inserm (Institut National De La Santè Et De La Recherche Médicale) | Methods for the treatment of epilepsy |
| BR112022002307A2 (pt) | 2019-08-09 | 2022-06-28 | Univ Massachusetts | Oligonucleotídeos quimicamente modificados que têm como alvo snps |
| WO2021053018A1 (en) | 2019-09-16 | 2021-03-25 | Uniqure Ip B.V. | Targeting misspliced transcripts in genetic disorders |
| CN114650822A (zh) | 2019-11-01 | 2022-06-21 | 诺华股份有限公司 | 剪接调节子用于减慢亨廷顿氏病进展的治疗的用途 |
| KR20220094213A (ko) * | 2019-11-01 | 2022-07-05 | 알닐람 파마슈티칼스 인코포레이티드 | 헌팅틴 (HTT) iRNA 제제 조성물 및 이의 사용 방법 |
| WO2021204872A1 (en) * | 2020-04-07 | 2021-10-14 | Uniqure Ip B.V. | Gene constructs for silencing angiopoietin-like 3 (angptl3) and uses thereof |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| US20220090069A1 (en) * | 2020-08-03 | 2022-03-24 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
| TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
| AU2022246786A1 (en) | 2021-03-29 | 2023-10-05 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| CN115141848A (zh) * | 2021-03-30 | 2022-10-04 | 南京大学 | 一种用于治疗亨廷顿病的rna递送系统 |
| EP4359525A1 (en) | 2021-06-21 | 2024-05-01 | uniQure biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| JP2025503910A (ja) * | 2022-01-27 | 2025-02-06 | アスクレピオス バイオファーマシューティカル， インコーポレイテッド | 神経疾患を処置するための組成物 |
| EP4508213A1 (en) | 2022-04-12 | 2025-02-19 | uniQure biopharma B.V. | Nucleic acid regulation of apoe |
| EP4508214A1 (en) | 2022-04-12 | 2025-02-19 | uniQure biopharma B.V. | Nucleic acid regulation of c9orf72 |
| US20250230437A1 (en) | 2022-04-12 | 2025-07-17 | Uniqure Biopharma B.V. | Novel systems for nucleic acid regulation |
| WO2023198663A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Nucleic acid regulation of snca |
| WO2024218204A1 (en) | 2023-04-18 | 2024-10-24 | Uniqure Biopharma B.V. | Gene delivery vehicles comprising rna and antibodies |
| WO2025051805A1 (en) | 2023-09-04 | 2025-03-13 | Uniqure Biopharma B.V. | Novel neurotropic recombinant adeno-associated virus particles |
| WO2025078598A1 (en) | 2023-10-11 | 2025-04-17 | Uniqure Biopharma B.V. | Novel nucleic acid regulation of huntingtin gene |
| WO2025078602A1 (en) | 2023-10-11 | 2025-04-17 | Uniqure Biopharma B.V. | Nucleic acid regulation of msh3 in repeat expansion disorders |
| WO2025078606A1 (en) | 2023-10-11 | 2025-04-17 | Uniqure Biopharma B.V. | Further novel systems for nucleic acid regulation |
| WO2025106739A1 (en) * | 2023-11-14 | 2025-05-22 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of htt expression |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2526893C (en) | 2003-05-14 | 2010-10-26 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| EP1735443A2 (en) | 2004-04-14 | 2006-12-27 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| CN101287837B (zh) | 2005-10-20 | 2015-03-18 | 尤尼克尔生物制药股份有限公司 | 昆虫细胞中生产的改进的aav载体 |
| WO2007148971A2 (en) | 2006-06-21 | 2007-12-27 | Amsterdam Molecular Therapeutics B.V. | Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells |
| CA2662704A1 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
| US20090036395A1 (en) * | 2007-04-26 | 2009-02-05 | Davidson Beverly L | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| PT2164967E (pt) | 2007-05-31 | 2015-10-27 | Univ Iowa Res Found | Redução da toxicidade não dirigida do arn de interferência |
| PL3093345T3 (pl) | 2007-07-26 | 2020-03-31 | Uniqure Ip B.V. | Wektory bakulowirusowe zawierające powtórzone sekwencje kodujące o zróżnicowanej preferencji kodonów |
| EP2250256B1 (en) | 2008-02-19 | 2017-08-02 | uniQure IP B.V. | Optimisation of expression of parvoviral rep and cap proteins in insect cells |
| EP2411505A4 (en) * | 2009-03-26 | 2013-01-30 | Univ California | MESENCHYMAL STEM CELLS FOR PRODUCING HEMDERING RNA FOR SICKNESS MODIFICATION |
| HRP20181423T1 (hr) * | 2009-05-02 | 2018-11-30 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| WO2011122950A1 (en) | 2010-04-01 | 2011-10-06 | Amsterdam Molecular Therapeutics (Amt) Ip B.V. | Monomeric duplex aav vectors |
| EP2561078B1 (en) * | 2010-04-23 | 2018-09-19 | Cold Spring Harbor Laboratory | NOVEL STRUCTURALLY DESIGNED shRNAs |
| EP2583692A4 (en) * | 2010-06-18 | 2014-11-26 | Lsip Llc | HEMMER OF THE EXPRESSION OF DOMINANT ALLELES |
| KR101961347B1 (ko) | 2011-09-08 | 2019-03-25 | 유니큐어 아이피 비.브이. | Aav 제조물로부터 오염 바이러스의 제거 |
| CN103224556B (zh) * | 2013-01-31 | 2015-04-15 | 清华大学 | 亨廷顿舞蹈症的靶位蛋白质及其编码基因和应用 |
| WO2016102664A1 (en) * | 2014-12-24 | 2016-06-30 | Uniqure Ip B.V. | Rnai induced huntingtin gene suppression |
-
2015
- 2015-12-23 WO PCT/EP2015/081157 patent/WO2016102664A1/en active Application Filing
- 2015-12-23 PL PL15817385T patent/PL3237618T3/pl unknown
- 2015-12-23 TR TR2019/08969T patent/TR201908969T4/tr unknown
- 2015-12-23 PT PT15817385T patent/PT3237618T/pt unknown
- 2015-12-23 HU HUE15817385A patent/HUE043842T2/hu unknown
- 2015-12-23 SI SI201530791T patent/SI3237618T1/sl unknown
- 2015-12-23 HR HRP20190992TT patent/HRP20190992T1/hr unknown
- 2015-12-23 AU AU2015370903A patent/AU2015370903B2/en active Active
- 2015-12-23 EA EA201791417A patent/EA037696B1/ru unknown
- 2015-12-23 US US15/538,964 patent/US10174321B2/en active Active
- 2015-12-23 HK HK18105533.3A patent/HK1246344B/en unknown
- 2015-12-23 MX MX2017008500A patent/MX381121B/es unknown
- 2015-12-23 LT LTEP15817385.6T patent/LT3237618T/lt unknown
- 2015-12-23 ES ES15817385T patent/ES2732023T3/es active Active
- 2015-12-23 EP EP19167349.0A patent/EP3540063A1/en active Pending
- 2015-12-23 JP JP2017552234A patent/JP6839094B2/ja active Active
- 2015-12-23 DK DK15817385.6T patent/DK3237618T3/da active
- 2015-12-23 CN CN201580071228.2A patent/CN108064292B/zh active Active
- 2015-12-23 KR KR1020177020454A patent/KR102636633B1/ko active Active
- 2015-12-23 CA CA2971920A patent/CA2971920C/en active Active
- 2015-12-23 EP EP15817385.6A patent/EP3237618B1/en active Active
- 2015-12-23 SM SM20190347T patent/SMT201900347T1/it unknown
- 2015-12-23 RS RS20190744A patent/RS58862B1/sr unknown
-
2017
- 2017-06-18 IL IL252990A patent/IL252990B/en unknown
- 2017-07-24 CO CONC2017/0007335A patent/CO2017007335A2/es unknown
-
2018
- 2018-11-16 US US16/193,908 patent/US10767180B2/en active Active
-
2019
- 2019-06-13 CY CY20191100616T patent/CY1121733T1/el unknown
-
2020
- 2020-07-15 US US16/930,035 patent/US11371044B2/en active Active
- 2020-10-01 JP JP2020167128A patent/JP2021000136A/ja active Pending
-
2022
- 2022-05-24 US US17/751,934 patent/US20230119344A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CY1121733T1 (el) | Επαγομενη απο rnai καταστολη του γονιδιου της χαντινγκτινης | |
| CY1125364T1 (el) | Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου ηαο1 (οξειδαση υδροξυοξεος 1 (γλυκολικη οξειδαση) | |
| Gazave et al. | Posterior elongation in the annelid Platynereis dumerilii involves stem cells molecularly related to primordial germ cells | |
| CY1125029T1 (el) | Τροποποιημενοι νουκλεοζιτες, νουκλεοτιδια, και νουκλεϊκα οξεα και χρησεις αυτων | |
| CY1121322T1 (el) | Αντινοηματικο νουκλειϊκο οξυ | |
| IL269431A (en) | Methods for targeted nucleic acid sequence enrichment with applications to error corrected nucleic acid sequencing | |
| MX379233B (es) | Métodos y composiciones para el perfilado de adn. | |
| MX2022007933A (es) | Compuestos de polimetino y su uso como etiquetas fluorescentes. | |
| PE20191351A1 (es) | Tratamiento aav de la enfermedad de huntington | |
| CO2017000357A2 (es) | Ácidos nucleicos antisentido | |
| FI3604532T3 (fi) | Uusia crispr-entsyymejä ja järjestelmiä | |
| MX2015016798A (es) | Regulacion transcripcional guiada por acido ribonucleico. | |
| MX2016007541A (es) | Moléculas modificadas de ácido nucleico y usos de las mismas. | |
| EP4028512A4 (en) | Modified bacterial retroelement with enhanced dna production | |
| JP2017502668A5 (el) | ||
| MX379058B (es) | Metodo para analisis de metilacion. | |
| UY36189A (es) | Composiciones y métodos para regular la expresión génica por medio de la interferencia de arn | |
| MY196912A (en) | Short pendant arm linkers for nucleotides in sequencing applications | |
| IL277813A (en) | Tm-enhanced double-helix nucleic acid tracer molecules with nucleotides | |
| CO2018000194A2 (es) | Alelos de dominio mads-box para controlar el fenotipo de membrana en la palma | |
| HK1259060A1 (zh) | 抑制腎素原基因或腎素原受體基因表達的單鏈核酸分子及其用途 | |
| CL2021002842A1 (es) | Moléculas inhibidoras de ácido nucleico bicatenario con cadenas de sentido acortadas | |
| WO2016106387A3 (en) | Nucleic acid aptamers to treat histone-induced disease states | |
| PH12017501090A1 (en) | Parental rnai suppression of kruppel gene to control coleopteran pests | |
| EP3418289A4 (en) | ARTIFICIAL NUCLEOSIDE AND ARTIFICIAL NUCLEOTIDE AND ARTIFICIAL OLIGONUCLEOTIDE |