JP2013529181A5 - - Google Patents
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- JP2013529181A5 JP2013529181A5 JP2013501487A JP2013501487A JP2013529181A5 JP 2013529181 A5 JP2013529181 A5 JP 2013529181A5 JP 2013501487 A JP2013501487 A JP 2013501487A JP 2013501487 A JP2013501487 A JP 2013501487A JP 2013529181 A5 JP2013529181 A5 JP 2013529181A5
- Authority
- JP
- Japan
- Prior art keywords
- pkd1
- antibody
- human mammal
- mammal
- endogenous
- 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
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- 102100016976 PRKD1 Human genes 0.000 claims 20
- 101710025004 PRKD1 Proteins 0.000 claims 20
- 108090001123 antibodies Proteins 0.000 claims 8
- 102000004965 antibodies Human genes 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 150000007523 nucleic acids Chemical class 0.000 claims 6
- 241000124008 Mammalia Species 0.000 claims 5
- 239000000203 mixture Substances 0.000 claims 5
- 230000002452 interceptive Effects 0.000 claims 4
- 210000004027 cells Anatomy 0.000 claims 3
- 239000003795 chemical substances by application Substances 0.000 claims 3
- 230000000694 effects Effects 0.000 claims 3
- 210000002569 neurons Anatomy 0.000 claims 3
- 206010053643 Neurodegenerative disease Diseases 0.000 claims 2
- 108020004459 Small Interfering RNA Proteins 0.000 claims 2
- 229920001985 Small interfering RNA Polymers 0.000 claims 2
- 230000000051 modifying Effects 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 206010001897 Alzheimer's disease Diseases 0.000 claims 1
- 206010002026 Amyotrophic lateral sclerosis Diseases 0.000 claims 1
- 208000008208 Craniocerebral Trauma Diseases 0.000 claims 1
- 201000001971 Huntington's disease Diseases 0.000 claims 1
- 206010027175 Memory impairment Diseases 0.000 claims 1
- 208000009025 Nervous System Disease Diseases 0.000 claims 1
- 206010029305 Neurological disorder Diseases 0.000 claims 1
- 206010029331 Neuropathy peripheral Diseases 0.000 claims 1
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 1
- 206010061536 Parkinson's disease Diseases 0.000 claims 1
- 241000283984 Rodentia Species 0.000 claims 1
- 208000008513 Spinal Cord Injury Diseases 0.000 claims 1
- 208000006011 Stroke Diseases 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 229940079593 drugs Drugs 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 108010045030 monoclonal antibodies Proteins 0.000 claims 1
- 102000005614 monoclonal antibodies Human genes 0.000 claims 1
- 201000006417 multiple sclerosis Diseases 0.000 claims 1
- 210000003867 nerve cell Anatomy 0.000 claims 1
- 210000004255 neuroglia Anatomy 0.000 claims 1
- 201000001119 neuropathy Diseases 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 108091008117 polyclonal antibodies Proteins 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 150000003384 small molecules Chemical group 0.000 claims 1
- 241000894007 species Species 0.000 claims 1
Claims (20)
- 個体の神経障害または神経変性疾患を治療するための医薬組成物であって、前記個体の神経細胞および／またはグリア細胞内のプロテインキナーゼＤ１（ＰＫＤ１）の活性を調節する有効量の薬剤を含む組成物。
- 前記薬剤がＰＫＤ１の小分子モジュレーターである、請求項１に記載の組成物。
- 前記薬剤が神経細胞内の活性のあるＰＫＤ１のレベルを低下させる抑制性核酸である、請求項１に記載の組成物。
- 前記薬剤が特異的にＰＫＤ１に結合する抗体である、請求項１に記載の組成物。
- 前記神経変性疾患が、パーキンソン病、ハンチントン病、筋萎縮性側索硬化症、多発性硬化症またはアルツハイマー病である、請求項１に記載の組成物。
- 前記神経障害が脳卒中、脊髄損傷、または頭部外傷である、請求項１に記載の組成物。
- 細胞内のプロテインキナーゼＤ１（ＰＫＤ１）のレベルを特異的に低下させる単離された干渉核酸。
- 前記干渉核酸が低分子干渉ＲＮＡ（ｓｉＲＮＡ）である、請求項７に記載の干渉核酸。
- 請求項７に記載の干渉核酸をコードするヌクレオチド配列を含む組換えコンストラクト。
- プロテインキナーゼＤ１（ＰＫＤ１）に特異的に結合する単離された抗体。
- 前記抗体がポリクローナル抗体である、請求項１０に記載の抗体。
- 前記抗体がモノクローナル抗体である、請求項１０に記載の抗体。
- 前記抗体が、少なくとも約１０−７Ｍの親和性でＰＫＤ１ポリペプチドに結合する、請求項１０に記載の抗体。
- 内在性プロテインキナーゼＤ１（ＰＫＤ１）遺伝子に欠陥を含む遺伝子組換え非ヒト哺乳動物であって、前記遺伝子組換え非ヒト哺乳動物が、前記内在性ＰＫＤ１遺伝子に安定的に組み込まれた異種核酸を含み、その結果、前記遺伝子組換え非ヒト哺乳動物が、同じ種の対照哺乳動物におけるＰＫＤ１活性レベルと比較して、ＰＫＤ１活性レベルを減少させるという欠陥が、前記内在性ＰＫＤ１遺伝子にもたらされる、遺伝子組換え非ヒト哺乳動物。
- 前記哺乳動物が、内在性ＰＫＤ１遺伝子欠損についてヘテロ接合である、請求項１４に記載の遺伝子組換え非ヒト哺乳動物。
- 前記哺乳動物が、内在性ＰＫＤ１遺伝子欠損についてホモ接合である、請求項１４に記載の遺伝子組換え非ヒト哺乳動物。
- 前記哺乳動物が齧歯類である、請求項１４に記載の遺伝子組換え非ヒト哺乳動物。
- 前記哺乳動物が、空間記憶障害を呈する、請求項１４に記載の遺伝子組換え非ヒト哺乳動物。
- 内在性ＰＫＤ１遺伝子の欠陥が神経細胞特異的である、請求項１４に記載の遺伝子組換え非ヒト哺乳動物。
- 請求項１４に記載の遺伝子組換え非ヒト哺乳動物から単離された細胞であって、破壊された内在性ＰＫＤ１遺伝子を有する細胞。
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US31759810P | 2010-03-25 | 2010-03-25 | |
| US61/317,598 | 2010-03-25 | ||
| PCT/US2011/029806 WO2011119842A1 (en) | 2010-03-25 | 2011-03-24 | Compositions and methods for treating neurological disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2013529181A JP2013529181A (ja) | 2013-07-18 |
| JP2013529181A5 true JP2013529181A5 (ja) | 2014-05-08 |
Family
ID=44673620
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013501487A Pending JP2013529181A (ja) | 2010-03-25 | 2011-03-24 | 神経障害を治療するための組成物および方法 |
Country Status (4)
| Country | Link |
|---|---|
| US (2) | US9359445B2 (ja) |
| EP (1) | EP2550361B1 (ja) |
| JP (1) | JP2013529181A (ja) |
| WO (1) | WO2011119842A1 (ja) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3030902B1 (en) * | 2013-08-07 | 2019-09-25 | Friedrich Miescher Institute for Biomedical Research | New screening method for the treatment friedreich's ataxia |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| JP6867945B2 (ja) | 2014-10-03 | 2021-05-12 | コールド スプリング ハーバー ラボラトリー | 核内遺伝子出力の標的とされた増強 |
| WO2017060731A1 (en) | 2015-10-09 | 2017-04-13 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| EP3933041B1 (en) | 2015-12-14 | 2024-01-31 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant retardation |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| WO2018148365A1 (en) * | 2017-02-09 | 2018-08-16 | Cognoa, Inc. | Platform and system for digital personalized medicine |
| JP6985684B2 (ja) * | 2017-02-16 | 2021-12-22 | 国立大学法人京都大学 | 細胞評価方法、細胞評価装置、及び細胞評価プログラム |
| PL3673080T3 (pl) | 2017-08-25 | 2024-03-11 | Stoke Therapeutics, Inc. | Oligomery antysensowne do leczenia stanów i chorób |
| KR20230022409A (ko) | 2020-05-11 | 2023-02-15 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머 |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2093664C (en) | 1990-10-12 | 2003-07-29 | Fritz Eckstein | Modified ribozymes |
| DE4216134A1 (de) | 1991-06-20 | 1992-12-24 | Europ Lab Molekularbiolog | Synthetische katalytische oligonukleotidstrukturen |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| CA2154363A1 (en) | 1993-01-22 | 1994-08-04 | Bruce A. Sullenger | Localization of therapeutic agents |
| US5627053A (en) | 1994-03-29 | 1997-05-06 | Ribozyme Pharmaceuticals, Inc. | 2'deoxy-2'-alkylnucleotide containing nucleic acid |
| US5716824A (en) | 1995-04-20 | 1998-02-10 | Ribozyme Pharmaceuticals, Inc. | 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes) |
| US7964598B2 (en) | 1995-10-17 | 2011-06-21 | The J. David Gladstone Institutes | ApoE4 domain interaction inhibitors and methods of use thereof |
| DE69637256T2 (de) | 1996-01-16 | 2008-06-19 | Sirna Therapeutics, Inc., Boulder | Synthese von Methoxynukleoside und enzymatische Nukleisäure Moleküle |
| US5849902A (en) | 1996-09-26 | 1998-12-15 | Oligos Etc. Inc. | Three component chimeric antisense oligonucleotides |
| DE19650758C1 (de) * | 1996-12-06 | 1998-01-02 | Deutsches Krebsforsch | PKD1-Fragmente mit Bindungsregionen für PKD1-spezifische Antikörper |
| US6124272A (en) * | 1999-04-09 | 2000-09-26 | Isis Pharmaceutical Inc. | Antisense modulation of PDK-1 expression |
| US7491805B2 (en) | 2001-05-18 | 2009-02-17 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
| US20050233329A1 (en) | 2002-02-20 | 2005-10-20 | Sirna Therapeutics, Inc. | Inhibition of gene expression using duplex forming oligonucleotides |
| US20050282188A1 (en) | 2001-05-18 | 2005-12-22 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA) |
| EP1627061B1 (en) | 2001-05-18 | 2009-08-12 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) |
| GB0117326D0 (en) | 2001-07-16 | 2001-09-05 | Univ Aberdeen | Napthoquinone-type inhibitors of protein aggregation |
| AR036604A1 (es) | 2001-09-21 | 2004-09-22 | Mitsubishi Pharma Corp | Derivados de 4-pirimidona 3-sustituida |
| US20040121407A1 (en) * | 2002-09-06 | 2004-06-24 | Elixir Pharmaceuticals, Inc. | Regulation of the growth hormone/IGF-1 axis |
| EP2065383A1 (en) | 2003-11-19 | 2009-06-03 | Signal Pharmaceuticals, Inc. | Indazole compounds and methods of use thereof as protein kinase inhibitors |
| BRPI0611863B1 (pt) | 2005-06-22 | 2021-11-23 | Plexxikon, Inc | Composto, bem como composição e kit compreendendo o mesmo, composto intermediário na preparação do mesmo, método para tratamento e uso do mesmo |
| GB0602178D0 (en) * | 2006-02-03 | 2006-03-15 | Merck Sharp & Dohme | Therapeutic treatment |
| US20090247519A1 (en) | 2006-04-26 | 2009-10-01 | Tony Michael Raynham | Amino-ethyl-amino-aryl (aeaa) compounds and their use |
| US20090175852A1 (en) | 2006-06-06 | 2009-07-09 | Schering Corporation | Imidazopyrazines as protein kinase inhibitors |
| US8240498B2 (en) | 2006-10-31 | 2012-08-14 | Crown Packaging Technology, Inc. | Resealable closure |
| US8618063B2 (en) * | 2008-04-09 | 2013-12-31 | The Board Of Trustees Of The University Of Illinois | Method for treating a synucleinopathy |
| WO2009147246A1 (en) * | 2008-06-06 | 2009-12-10 | Medizinische Universität Graz | Compounds reducing or inhibiting the expression of pkd1 for diagnosis and therapy of brain tumors |
| ES2385276B1 (es) | 2010-02-25 | 2013-07-05 | Universidad Del País Vasco | Compuestos para el tratamiento de alzheimer. |
-
2011
- 2011-03-24 WO PCT/US2011/029806 patent/WO2011119842A1/en active Application Filing
- 2011-03-24 JP JP2013501487A patent/JP2013529181A/ja active Pending
- 2011-03-24 EP EP11760223.5A patent/EP2550361B1/en not_active Not-in-force
-
2012
- 2012-09-24 US US13/625,597 patent/US9359445B2/en not_active Expired - Fee Related
-
2016
- 2016-05-05 US US15/147,814 patent/US20160354372A1/en not_active Abandoned
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