CN109952114A - 用于治疗醛脱氢酶缺乏的基因疗法 - Google Patents

用于治疗醛脱氢酶缺乏的基因疗法 Download PDF

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Publication number
CN109952114A
CN109952114A CN201780058722.4A CN201780058722A CN109952114A CN 109952114 A CN109952114 A CN 109952114A CN 201780058722 A CN201780058722 A CN 201780058722A CN 109952114 A CN109952114 A CN 109952114A
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China
Prior art keywords
carrier
aldehyde dehydrogenase
mammal
aldh2
ala
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Pending
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CN201780058722.4A
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English (en)
Chinese (zh)
Inventor
M·加斯米
R·G·克雷斯托
O·E·帕格维齐
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Cornell University
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Cornell University
Adverum Biotechnologies Inc
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Publication of CN109952114A publication Critical patent/CN109952114A/zh
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    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
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    • C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
    • C12Y101/01001—Alcohol dehydrogenase (1.1.1.1)
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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CN201780058722.4A 2016-07-26 2017-07-26 用于治疗醛脱氢酶缺乏的基因疗法 Pending CN109952114A (zh)

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

Publications (1)

Publication Number Publication Date
CN109952114A true CN109952114A (zh) 2019-06-28

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CN201780058722.4A Pending CN109952114A (zh) 2016-07-26 2017-07-26 用于治疗醛脱氢酶缺乏的基因疗法

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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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114891808A (zh) * 2022-06-21 2022-08-12 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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
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
GB202218090D0 (en) * 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130267501A1 (en) * 2008-10-28 2013-10-10 The Board Of Trustees Of The Leland Stanford Junior University Modulators of Aldehyde Dehydrogenase and Methods of Use Thereof
US20140221462A1 (en) * 2013-02-01 2014-08-07 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia

Family Cites Families (8)

* Cited by examiner, † Cited by third party
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
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
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130267501A1 (en) * 2008-10-28 2013-10-10 The Board Of Trustees Of The Leland Stanford Junior University Modulators of Aldehyde Dehydrogenase and Methods of Use Thereof
US20140221462A1 (en) * 2013-02-01 2014-08-07 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
FRANC¸ OISE PIGUET，等: "Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice", 《HUMAN GENE THERAPY》 *
STEFANIE HAUG Æ MARKUS BRAUN-FALCO: "Adeno-associated virus vectors are able to restore fatty aldehyde dehydrogenase-deficiency. Implications for gene therapy in Sjo¨ gren-Larsson syndrome", 《ARCH DERMATOL RES》 *
X.Y. HU，等: "Aldehyde dehydrogenase 2 protects human umbilical vein endothelial cells against oxidative damage and increases endothelial nitric oxide production to reverse nitroglycerin tolerance", 《GENETICS AND MOLECULAR RESEARCH》 *
YUKI MATSUMURA，等: "Gene Therapy Correction of Aldehyde Dehydrogenase 2 Deficiency", 《MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114891808A (zh) * 2022-06-21 2022-08-12 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
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

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SG10202100632WA (en) 2021-03-30
AU2017301819B2 (en) 2024-09-26
EP3490613A4 (en) 2020-03-04
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
JP7165357B2 (ja) 2022-11-04
JP2019521692A (ja) 2019-08-08
EP3490613A1 (en) 2019-06-05
JP2022116045A (ja) 2022-08-09
KR102621134B1 (ko) 2024-01-04

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