CN117835986A - 使用双链siRNA进行小胶质基因沉默 - Google Patents
使用双链siRNA进行小胶质基因沉默 Download PDFInfo
- Publication number
- CN117835986A CN117835986A CN202280036538.0A CN202280036538A CN117835986A CN 117835986 A CN117835986 A CN 117835986A CN 202280036538 A CN202280036538 A CN 202280036538A CN 117835986 A CN117835986 A CN 117835986A
- Authority
- CN
- China
- Prior art keywords
- nucleotides
- length
- gene
- sense strand
- antisense strand
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
-
- 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
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- 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
- C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
-
- 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
- C12N2310/52—Physical structure branched
-
- 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
- C12N2320/31—Combination therapy
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163165518P | 2021-03-24 | 2021-03-24 | |
| US63/165,518 | 2021-03-24 | ||
| PCT/US2022/021789 WO2022204429A1 (en) | 2021-03-24 | 2022-03-24 | Microglial gene silencing using double-stranded sirna |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN117835986A true CN117835986A (zh) | 2024-04-05 |
Family
ID=83397877
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202280036538.0A Pending CN117835986A (zh) | 2021-03-24 | 2022-03-24 | 使用双链siRNA进行小胶质基因沉默 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240200063A1 (https=) |
| EP (1) | EP4313070A4 (https=) |
| JP (1) | JP2024512612A (https=) |
| CN (1) | CN117835986A (https=) |
| AU (1) | AU2022246147A1 (https=) |
| CA (1) | CA3214657A1 (https=) |
| WO (1) | WO2022204429A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN118374495A (zh) * | 2024-03-28 | 2024-07-23 | 深圳脑健生物技术有限公司 | 一种抑制NLRP3基因表达的siRNA及其前体和应用 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2006055727A2 (en) * | 2004-11-18 | 2006-05-26 | The Board Of Trustees Of The University Of Illinois | MULTICISTRONIC siRNA CONSTRUCTS TO INHIBIT TUMORS |
| SMT202000454T1 (it) * | 2015-04-03 | 2020-11-10 | Univ Massachusetts | Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina |
| WO2017132669A1 (en) * | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
| US20200385737A1 (en) * | 2019-03-29 | 2020-12-10 | University Of Massachusetts | OLIGONUCLEOTIDE-BASED MODULATION OF C9orf72 |
| WO2021026490A1 (en) * | 2019-08-08 | 2021-02-11 | Mpeg La, L.L.C. | Cns targeting with multimeric oligonucleotides |
| MX2022001710A (es) * | 2019-08-09 | 2022-05-10 | Univ Massachusetts | Oligonucleótidos modificados químicamente dirigidos a los snp. |
-
2022
- 2022-03-24 EP EP22776672.2A patent/EP4313070A4/en active Pending
- 2022-03-24 US US18/283,671 patent/US20240200063A1/en active Pending
- 2022-03-24 JP JP2023558983A patent/JP2024512612A/ja active Pending
- 2022-03-24 CN CN202280036538.0A patent/CN117835986A/zh active Pending
- 2022-03-24 AU AU2022246147A patent/AU2022246147A1/en active Pending
- 2022-03-24 CA CA3214657A patent/CA3214657A1/en active Pending
- 2022-03-24 WO PCT/US2022/021789 patent/WO2022204429A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| CA3214657A1 (en) | 2022-09-29 |
| EP4313070A1 (en) | 2024-02-07 |
| AU2022246147A1 (en) | 2023-11-09 |
| US20240200063A1 (en) | 2024-06-20 |
| EP4313070A4 (en) | 2025-06-11 |
| WO2022204429A1 (en) | 2022-09-29 |
| JP2024512612A (ja) | 2024-03-19 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20240182892A1 (en) | Double-stranded sirna having patterned chemical modifications | |
| US20240287526A1 (en) | Compositions and methods for delivering therapeutic oligonucleotides to the central nervous system | |
| JP2025531482A (ja) | 微小管結合タンパク質タウ核酸を標的化するsiRNA組成物及び方法 | |
| US20240200063A1 (en) | Microglial gene silencing using double-stranded sirna | |
| US20250109402A1 (en) | Compositions and methods for treatment of pain | |
| WO2026026776A1 (zh) | 抑制APP基因表达的siRNA及其缀合物和应用 | |
| JP2025532885A (ja) | 神経変性疾患の処置のための組成物及び方法 | |
| JP2025531483A (ja) | 神経炎症疾患の治療のための組成物及び方法 | |
| US20260098264A1 (en) | Compositions and methods for treatment of neurological disorders | |
| US20250243492A1 (en) | Compositions and methods for treatment of prion diseases | |
| US20260098269A1 (en) | Compositions and methods for treatment of neuroinflammatory diseases | |
| JP2025531481A (ja) | 脊髄小脳変性症の治療のための組成物及び方法 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PB01 | Publication | ||
| PB01 | Publication | ||
| SE01 | Entry into force of request for substantive examination | ||
| SE01 | Entry into force of request for substantive examination | ||
| CB03 | Change of inventor or designer information |
Inventor after: D cutis Inventor after: A. Jackson Inventor after: B. Andean Inventor after: B. M.D.C. Godingho Inventor after: Chen Qingmin Inventor before: D cutis Inventor before: A. Jackson Inventor before: B. Andean Inventor before: B. Godingho Inventor before: Chen Qingmin |
|
| CB03 | Change of inventor or designer information |