JP2019521692A5 - - Google Patents
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- Publication number
- JP2019521692A5 JP2019521692A5 JP2019504068A JP2019504068A JP2019521692A5 JP 2019521692 A5 JP2019521692 A5 JP 2019521692A5 JP 2019504068 A JP2019504068 A JP 2019504068A JP 2019504068 A JP2019504068 A JP 2019504068A JP 2019521692 A5 JP2019521692 A5 JP 2019521692A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- composition
- aldehyde dehydrogenase
- mammal
- vector according
- 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.)
- Granted
Links
- 239000013598 vector Substances 0.000 claims 27
- 102000005369 Aldehyde Dehydrogenase Human genes 0.000 claims 9
- 108020002663 Aldehyde Dehydrogenase Proteins 0.000 claims 9
- 241000124008 Mammalia Species 0.000 claims 8
- 230000007812 deficiency Effects 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 150000007523 nucleic acids Chemical group 0.000 claims 3
- 239000013607 AAV vector Substances 0.000 claims 2
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- 239000013603 viral vector Substances 0.000 claims 2
- 108700028369 Alleles Proteins 0.000 claims 1
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- 206010017993 Gastrointestinal neoplasms Diseases 0.000 claims 1
- 206010020772 Hypertension Diseases 0.000 claims 1
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 1
- 239000004472 Lysine Substances 0.000 claims 1
- 241000282560 Macaca mulatta Species 0.000 claims 1
- 102000009645 Mitochondrial Aldehyde Dehydrogenase Human genes 0.000 claims 1
- 108010009513 Mitochondrial Aldehyde Dehydrogenase Proteins 0.000 claims 1
- SNIOPGDIGTZGOP-UHFFFAOYSA-N Nitroglycerin Chemical compound [O-][N+](=O)OCC(O[N+]([O-])=O)CO[N+]([O-])=O SNIOPGDIGTZGOP-UHFFFAOYSA-N 0.000 claims 1
- 239000000006 Nitroglycerin Substances 0.000 claims 1
- 208000001132 Osteoporosis Diseases 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 208000006011 Stroke Diseases 0.000 claims 1
- 125000000539 amino acid group Chemical group 0.000 claims 1
- 208000007502 anemia Diseases 0.000 claims 1
- 206010003119 arrhythmia Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims 1
- 229960003711 glyceryl trinitrate Drugs 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 238000001361 intraarterial administration Methods 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000007912 intraperitoneal administration Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 230000002438 mitochondrial effect Effects 0.000 claims 1
- 208000010125 myocardial infarction Diseases 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 230000000241 respiratory effect Effects 0.000 claims 1
- 230000001177 retroviral effect Effects 0.000 claims 1
- 206010041823 squamous cell carcinoma Diseases 0.000 claims 1
- 230000001988 toxicity Effects 0.000 claims 1
- 231100000419 toxicity Toxicity 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022078487A JP7558535B2 (ja) | 2016-07-26 | 2022-05-11 | アルデヒドデヒドロゲナーゼ欠損症の治療のための遺伝子治療 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662367012P | 2016-07-26 | 2016-07-26 | |
| US62/367,012 | 2016-07-26 | ||
| PCT/US2017/043999 WO2018022783A1 (en) | 2016-07-26 | 2017-07-26 | Gene therapy for the treatment of aldehyde dehydrogenase deficiency |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022078487A Division JP7558535B2 (ja) | 2016-07-26 | 2022-05-11 | アルデヒドデヒドロゲナーゼ欠損症の治療のための遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019521692A JP2019521692A (ja) | 2019-08-08 |
| JP2019521692A5 true JP2019521692A5 (enExample) | 2020-09-03 |
| JP7165357B2 JP7165357B2 (ja) | 2022-11-04 |
Family
ID=61016742
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019504068A Active JP7165357B2 (ja) | 2016-07-26 | 2017-07-26 | アルデヒドデヒドロゲナーゼ欠損症の治療のための遺伝子治療 |
| JP2022078487A Active JP7558535B2 (ja) | 2016-07-26 | 2022-05-11 | アルデヒドデヒドロゲナーゼ欠損症の治療のための遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022078487A Active JP7558535B2 (ja) | 2016-07-26 | 2022-05-11 | アルデヒドデヒドロゲナーゼ欠損症の治療のための遺伝子治療 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20190160187A1 (enExample) |
| EP (1) | EP3490613A4 (enExample) |
| JP (2) | JP7165357B2 (enExample) |
| KR (1) | KR102621134B1 (enExample) |
| CN (1) | CN109952114A (enExample) |
| AU (1) | AU2017301819B2 (enExample) |
| SG (2) | SG10202100632WA (enExample) |
| WO (1) | WO2018022783A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3141332A1 (en) * | 2019-06-03 | 2020-12-10 | Institute For Cancer Research D/B/A/ The Research Institute Of Fox Chase Cancer Center | Adeno-associated virus vector delivery of cystathionine beta-synthase (cbs) enzyme for treating cbs deficiency |
| WO2021119142A1 (en) * | 2019-12-09 | 2021-06-17 | The Trustees Of Columbia University In The City Of New York | 3d organoids for personalized oral cancer therapy |
| CN111471660B (zh) * | 2020-03-12 | 2023-11-24 | 广州辉园苑医药科技有限公司 | 一种乙醛脱氢酶重组基因及其乳酸菌载体和应用 |
| KR102557375B1 (ko) * | 2020-12-16 | 2023-07-20 | 동국대학교 와이즈캠퍼스 산학협력단 | 미토콘드리아 전구서열 유래 펩타이드 및 그 용도 |
| CN114891808B (zh) * | 2022-06-21 | 2023-10-27 | 珠海丽凡达生物技术有限公司 | 编码ALDH2多肽的mRNA分子、应用及mRNA药物 |
| GB202218090D0 (en) * | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2004252559B2 (en) * | 2003-06-25 | 2010-07-29 | Gencia Corporation | Modified vectors for organelle transfection |
| WO2009137045A2 (en) * | 2008-05-07 | 2009-11-12 | The Board Of Trustees Of The Leland Stanford Junior University | Crystal structure of aldehyde dehydrogenase and methods of use thereof |
| WO2010062308A1 (en) * | 2008-10-28 | 2010-06-03 | The Board Of Trustees Of The Leland Stanford Junior University | Modulators of aldehyde dehydrogenase and methods of use thereof |
| EP3514232A1 (en) * | 2010-04-23 | 2019-07-24 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| JP6091435B2 (ja) * | 2011-02-22 | 2017-03-08 | カリフォルニア インスティチュート オブ テクノロジー | アデノ随伴ウイルス（ａａｖ）ベクターを用いたタンパク質の送達 |
| US9066966B2 (en) * | 2013-02-01 | 2015-06-30 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia |
| EP2988737B1 (en) * | 2013-04-23 | 2021-04-07 | Nizyme Inc. | Methods and compositions for treating diseases |
| ES2833157T3 (es) * | 2014-02-19 | 2021-06-14 | Aviv Therapeutics Inc | Amidas políciclicas de unión a aldehído deshidrogenasa 2 mitocondrial (aldh2) y su uso para el tratamiento del cáncer |
| EP3389724B1 (en) * | 2015-12-14 | 2020-07-01 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| JP7082050B2 (ja) * | 2015-12-14 | 2022-06-07 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 脊髄性筋萎縮症の処置において有用なアデノ－関連ウイルスベクター |
-
2017
- 2017-07-26 SG SG10202100632WA patent/SG10202100632WA/en unknown
- 2017-07-26 WO PCT/US2017/043999 patent/WO2018022783A1/en not_active Ceased
- 2017-07-26 KR KR1020197005649A patent/KR102621134B1/ko active Active
- 2017-07-26 US US16/321,023 patent/US20190160187A1/en active Pending
- 2017-07-26 EP EP17835222.5A patent/EP3490613A4/en active Pending
- 2017-07-26 CN CN201780058722.4A patent/CN109952114A/zh active Pending
- 2017-07-26 JP JP2019504068A patent/JP7165357B2/ja active Active
- 2017-07-26 SG SG11201900543SA patent/SG11201900543SA/en unknown
- 2017-07-26 AU AU2017301819A patent/AU2017301819B2/en active Active
-
2022
- 2022-05-11 JP JP2022078487A patent/JP7558535B2/ja active Active
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