JP7165357B2 - 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 - Google Patents

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 Download PDF

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JP7165357B2
JP7165357B2 JP2019504068A JP2019504068A JP7165357B2 JP 7165357 B2 JP7165357 B2 JP 7165357B2 JP 2019504068 A JP2019504068 A JP 2019504068A JP 2019504068 A JP2019504068 A JP 2019504068A JP 7165357 B2 JP7165357 B2 JP 7165357B2
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aldh2
aldehyde dehydrogenase
aav
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JP2019521692A5 (enExample
JP2019521692A (ja
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ガスミ、メディ
ジー． クリスタル、ロナルド
イー． パゴヴィッチ、オデリア
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コーネル ユニヴァーシティー
アドヴェラム バイオテクノロジーズ， インコーポレイテッド
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JP2019504068A 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 Active JP7165357B2 (ja)

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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

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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

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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

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US (1) US20190160187A1 (enExample)
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KR (1) KR102621134B1 (enExample)
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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

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007524392A (ja) 2003-06-25 2007-08-30 ゲンシア コーポレーション 細胞小器官トランスフェクションのための改質ベクター
JP2012506856A (ja) 2008-10-28 2012-03-22 ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティ アルデヒドデヒドロゲナーゼのモジュレーターおよびその使用方法
JP2013531471A (ja) 2010-04-23 2013-08-08 ユニバーシティ オブ マサチューセッツ Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2014511180A (ja) 2011-02-22 2014-05-15 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
WO2014176309A1 (en) 2013-04-23 2014-10-30 Nizyme, Inc. Methods and compositions for treating diseases
WO2015127137A1 (en) 2014-02-19 2015-08-27 Aldea Pharmaceuticals, Inc. Mitochondrial aldehyde dehydrogenase 2 (aldh2) binding polycyclic amides and their use for the treatment of cancer

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
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

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007524392A (ja) 2003-06-25 2007-08-30 ゲンシア コーポレーション 細胞小器官トランスフェクションのための改質ベクター
JP2012506856A (ja) 2008-10-28 2012-03-22 ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティ アルデヒドデヒドロゲナーゼのモジュレーターおよびその使用方法
JP2013531471A (ja) 2010-04-23 2013-08-08 ユニバーシティ オブ マサチューセッツ Ｃｎｓターゲティングａａｖベクターおよびその使用方法
JP2014511180A (ja) 2011-02-22 2014-05-15 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
WO2014176309A1 (en) 2013-04-23 2014-10-30 Nizyme, Inc. Methods and compositions for treating diseases
WO2015127137A1 (en) 2014-02-19 2015-08-27 Aldea Pharmaceuticals, Inc. Mitochondrial aldehyde dehydrogenase 2 (aldh2) binding polycyclic amides and their use for the treatment of cancer

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Arch Dematol Res，Vol.296，2005年，p.568-572
Genetics and molecular research，2016年，15(2),:gmr.15027822
Human gene therapy，2012年，Vol.23，p.903-914

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JP7558535B2 (ja) 2024-10-01
SG11201900543SA (en) 2019-02-27
WO2018022783A1 (en) 2018-02-01
KR20190038583A (ko) 2019-04-08
AU2017301819A1 (en) 2019-02-07
US20190160187A1 (en) 2019-05-30
JP2019521692A (ja) 2019-08-08
EP3490613A1 (en) 2019-06-05
JP2022116045A (ja) 2022-08-09
KR102621134B1 (ko) 2024-01-04
CN109952114A (zh) 2019-06-28

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