JPWO2019222354A5 - - Google Patents
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- Publication number
- JPWO2019222354A5 JPWO2019222354A5 JP2020563947A JP2020563947A JPWO2019222354A5 JP WO2019222354 A5 JPWO2019222354 A5 JP WO2019222354A5 JP 2020563947 A JP2020563947 A JP 2020563947A JP 2020563947 A JP2020563947 A JP 2020563947A JP WO2019222354 A5 JPWO2019222354 A5 JP WO2019222354A5
- Authority
- JP
- Japan
- Prior art keywords
- viral vector
- promoter
- nucleic acid
- interfering rna
- rna
- 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
- 229920002477 rna polymer Polymers 0.000 claims 24
- 230000003612 virological Effects 0.000 claims 19
- 230000002452 interceptive Effects 0.000 claims 14
- 150000007523 nucleic acids Chemical group 0.000 claims 11
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 9
- 239000002679 microRNA Substances 0.000 claims 9
- 229920001239 microRNA Polymers 0.000 claims 6
- 241000700605 Viruses Species 0.000 claims 4
- 239000004055 small Interfering RNA Substances 0.000 claims 4
- 102000007469 Actins Human genes 0.000 claims 3
- 108010085238 Actins Proteins 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 3
- 102000001577 C9orf72 Protein Human genes 0.000 claims 3
- 108010093772 C9orf72 Protein Proteins 0.000 claims 3
- 229920002459 Intron Polymers 0.000 claims 3
- 108020004388 MicroRNAs Proteins 0.000 claims 2
- 102100002855 PITX3 Human genes 0.000 claims 2
- 101700062181 PITX3 Proteins 0.000 claims 2
- 108020004454 Phosphoglycerate kinases Proteins 0.000 claims 2
- 102000011755 Phosphoglycerate kinases Human genes 0.000 claims 2
- 229920001891 Small hairpin RNA Polymers 0.000 claims 2
- 229920001985 Small interfering RNA Polymers 0.000 claims 2
- 230000000295 complement Effects 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 108091007167 primary miRNA Proteins 0.000 claims 2
- 241000432074 Adeno-associated virus Species 0.000 claims 1
- 210000003917 Chromosomes, Human Anatomy 0.000 claims 1
- 102100000789 DES Human genes 0.000 claims 1
- 108010044052 Desmin Proteins 0.000 claims 1
- 229920000665 Exon Polymers 0.000 claims 1
- 108020004391 Introns Proteins 0.000 claims 1
- 229920001320 Leader sequence (mRNA) Polymers 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 210000003205 Muscles Anatomy 0.000 claims 1
- 108010084498 Myosin Heavy Chains Proteins 0.000 claims 1
- 102000005604 Myosin Heavy Chains Human genes 0.000 claims 1
- 108010067385 Myosin Light Chains Proteins 0.000 claims 1
- 102000016349 Myosin Light Chains Human genes 0.000 claims 1
- 206010068871 Myotonic dystrophy Diseases 0.000 claims 1
- 102000030951 Phosphotransferases Human genes 0.000 claims 1
- 108091000081 Phosphotransferases Proteins 0.000 claims 1
- 108050006987 Poxvirus Proteins 0.000 claims 1
- 102000001253 Protein Kinases Human genes 0.000 claims 1
- 108060006633 Protein Kinases Proteins 0.000 claims 1
- 229920000401 Three prime untranslated region Polymers 0.000 claims 1
- 102000013534 Troponin C Human genes 0.000 claims 1
- 108010065978 Troponin C Proteins 0.000 claims 1
- 102000013394 Troponin I Human genes 0.000 claims 1
- 108010065729 Troponin I Proteins 0.000 claims 1
- 241000700618 Vaccinia virus Species 0.000 claims 1
- 238000000137 annealing Methods 0.000 claims 1
- 230000000747 cardiac effect Effects 0.000 claims 1
- CVSVTCORWBXHQV-UHFFFAOYSA-N creatine zwitterion Chemical compound NC(=[NH2+])N(C)CC([O-])=O CVSVTCORWBXHQV-UHFFFAOYSA-N 0.000 claims 1
- 201000010901 lateral sclerosis Diseases 0.000 claims 1
- 210000000663 muscle cells Anatomy 0.000 claims 1
- 101700083244 myoD Proteins 0.000 claims 1
- 230000003274 myotonic Effects 0.000 claims 1
- 210000002569 neurons Anatomy 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241000701447 unidentified baculovirus Species 0.000 claims 1
- 241001529453 unidentified herpesvirus Species 0.000 claims 1
- 241001430294 unidentified retrovirus Species 0.000 claims 1
Claims (20)
(a)ヒトDMPKのエクソン1~15のいずれか1つ内の、
(b)ヒトDMPKのイントロン1~14のいずれか1つ内の、
(c)ヒトDMPK内のエクソン-イントロン境界を含む、又は
(d)ヒトDMPKの5'UTR又は3'UTR内の、
セグメントの核酸配列に少なくとも85%相補的な核酸配列を有する、請求項2~6のいずれか1項に記載のウイルスベクター。 The part of each interfering RNA is
(a) Within any one of exons 1 to 15 of human DMPK ,
(b) Within any one of the human DMPK introns 1-14,
(c) Includes or contains an exon-intron boundary within the human DMPK
(d) Within the 5'UTR or 3'UTR of the human DMPK,
The viral vector according to any one of claims 2 to 6 , which has a nucleic acid sequence that is at least 85% complementary to the nucleic acid sequence of the segment.
(a)成熟miRNAをコードするプライマリーmiRNA(pri-miRNA)転写物、又は
(b)成熟miRNAをコードするpre-miRNA転写物、
を含む、請求項1~13のいずれか1項に記載のウイルスベクター。 The interfering RNA is short interfering RNA (siRNA), short hairpin RNA (shRNA), or microRNA (miRNA) , optionally the miRNA is U6 miRNA, and optionally the virus vector.
(a) Primary miRNA (pri-miRNA) transcripts encoding mature miRNAs, or
(b) pre-miRNA transcripts encoding mature miRNAs,
The viral vector according to any one of claims 1 to 13 , which comprises .
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862671769P | 2018-05-15 | 2018-05-15 | |
US62/671,769 | 2018-05-15 | ||
PCT/US2019/032423 WO2019222354A1 (en) | 2018-05-15 | 2019-05-15 | Compositions and methods for reducing spliceopathy and treating rna dominance disorders |
Publications (2)
Publication Number | Publication Date |
---|---|
JP2021522836A JP2021522836A (en) | 2021-09-02 |
JPWO2019222354A5 true JPWO2019222354A5 (en) | 2022-05-24 |
Family
ID=68540673
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2020563947A Pending JP2021522836A (en) | 2018-05-15 | 2019-05-15 | Compositions and methods for reducing splice opacity and treating RNA dominance disorders |
Country Status (15)
Country | Link |
---|---|
US (1) | US20210269825A1 (en) |
EP (1) | EP3793566A4 (en) |
JP (1) | JP2021522836A (en) |
KR (1) | KR20210010549A (en) |
CN (1) | CN112469421A (en) |
AU (1) | AU2019268346A1 (en) |
BR (1) | BR112020023298A2 (en) |
CA (1) | CA3098249A1 (en) |
CL (1) | CL2020002955A1 (en) |
CO (1) | CO2020015239A2 (en) |
MA (1) | MA51938B1 (en) |
MX (1) | MX2020012269A (en) |
PH (1) | PH12020551913A1 (en) |
SG (1) | SG11202011151VA (en) |
WO (1) | WO2019222354A1 (en) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023034870A2 (en) | 2021-09-01 | 2023-03-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing dmpk expression |
WO2023196862A1 (en) * | 2022-04-06 | 2023-10-12 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
WO2023220719A2 (en) * | 2022-05-13 | 2023-11-16 | University Of Washington | Method for treatment of myotonic dystrophy combining protein expression and rna interference vector delivery with tissue detargeting |
WO2024006770A1 (en) * | 2022-06-27 | 2024-01-04 | Astellas Gene Therapies, Inc. | Compositions and methods for the treatment of myotonic dystrophies |
Family Cites Families (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5977333A (en) * | 1992-02-06 | 1999-11-02 | Massachusetts Institute Of Technology | DNA sequence encoding the myotonic dystrophy gene and uses thereof |
US20050042646A1 (en) * | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
BR112017013573A2 (en) * | 2014-12-24 | 2018-03-06 | Uniqure Ip Bv | double stranded rna, dna sequence, expression cassette, gene therapy vector, and host cell. |
US11260073B2 (en) * | 2015-11-02 | 2022-03-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating C90RF72 |
SG10202102997UA (en) * | 2016-09-30 | 2021-04-29 | Regeneron Pharma | Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus |
-
2019
- 2019-05-15 US US17/054,474 patent/US20210269825A1/en active Pending
- 2019-05-15 CN CN201980047374.XA patent/CN112469421A/en active Pending
- 2019-05-15 MX MX2020012269A patent/MX2020012269A/en unknown
- 2019-05-15 BR BR112020023298-0A patent/BR112020023298A2/en not_active Application Discontinuation
- 2019-05-15 AU AU2019268346A patent/AU2019268346A1/en active Pending
- 2019-05-15 WO PCT/US2019/032423 patent/WO2019222354A1/en unknown
- 2019-05-15 MA MA51938A patent/MA51938B1/en unknown
- 2019-05-15 CA CA3098249A patent/CA3098249A1/en active Pending
- 2019-05-15 EP EP19803882.0A patent/EP3793566A4/en active Pending
- 2019-05-15 SG SG11202011151VA patent/SG11202011151VA/en unknown
- 2019-05-15 KR KR1020207036197A patent/KR20210010549A/en unknown
- 2019-05-15 JP JP2020563947A patent/JP2021522836A/en active Pending
-
2020
- 2020-11-10 PH PH12020551913A patent/PH12020551913A1/en unknown
- 2020-11-13 CL CL2020002955A patent/CL2020002955A1/en unknown
- 2020-12-03 CO CONC2020/0015239A patent/CO2020015239A2/en unknown
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