CN110290814A - 用于治疗威尔逊病的基因疗法 - Google Patents

用于治疗威尔逊病的基因疗法 Download PDF

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Publication number
CN110290814A
CN110290814A CN201780085376.9A CN201780085376A CN110290814A CN 110290814 A CN110290814 A CN 110290814A CN 201780085376 A CN201780085376 A CN 201780085376A CN 110290814 A CN110290814 A CN 110290814A
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raav
atp7b
copper
mice
aav8
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詹姆斯·M·威尔逊
詹妮·艾格尼丝·西德兰
拉克什曼安·戈文达萨米
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University of Pennsylvania Penn
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    • A—HUMAN NECESSITIES
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Environmental Sciences (AREA)
CN201780085376.9A 2016-12-30 2017-12-29 用于治疗威尔逊病的基因疗法 Pending CN110290814A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662440659P 2016-12-30 2016-12-30
US62/440,659 2016-12-30
US201762473656P 2017-03-20 2017-03-20
US62/473,656 2017-03-20
PCT/US2017/068919 WO2018126116A1 (en) 2016-12-30 2017-12-29 Gene therapy for treating wilson's disease

Publications (1)

Publication Number Publication Date
CN110290814A true CN110290814A (zh) 2019-09-27

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CN201780085376.9A Pending CN110290814A (zh) 2016-12-30 2017-12-29 用于治疗威尔逊病的基因疗法

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US (2) US11473106B2 (enExample)
EP (2) EP3562514A4 (enExample)
JP (1) JP7128190B2 (enExample)
KR (2) KR102863181B1 (enExample)
CN (1) CN110290814A (enExample)
BR (1) BR112019013245A2 (enExample)
CA (1) CA3048044A1 (enExample)
MX (1) MX2019007873A (enExample)
WO (1) WO2018126116A1 (enExample)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115216481A (zh) * 2021-04-21 2022-10-21 四川至善唯新生物科技有限公司 一种表达量提高的截短型atp7b基因及其应用
CN115216533A (zh) * 2022-06-30 2022-10-21 湖南家辉生物技术有限公司 一种用于诊断威尔逊病的生物标志物、扩增引物组、检测试剂及应用
CN117947035A (zh) * 2024-03-25 2024-04-30 上海凌医生物科技有限公司 一种增强基因表达的启动子
CN120457199A (zh) * 2023-03-06 2025-08-08 凌意(杭州)生物科技有限公司 截短的atp7b及其用途

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110290814A (zh) * 2016-12-30 2019-09-27 宾夕法尼亚州立大学托管会 用于治疗威尔逊病的基因疗法
BR112020010883A2 (pt) 2017-12-01 2020-11-10 Encoded Therapeutics, Inc. proteínas de ligação a dna engenheiradas
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
BR112020015798A2 (pt) 2018-02-01 2021-03-09 Homology Medicines, Inc. Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
WO2020072451A1 (en) * 2018-10-01 2020-04-09 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
JP7532360B2 (ja) * 2018-11-16 2024-08-13 エンコーデッド セラピューティクス， インコーポレイテッド ウィルソン病を処置するための組成物および方法
JP7531495B2 (ja) * 2019-01-04 2024-08-09 ウルトラジェニックス ファーマシューティカル インコーポレイテッド ウイルソン病を処置するための遺伝子治療構築物
SG11202113048SA (en) 2019-05-29 2021-12-30 Encoded Therapeutics Inc Compositions and methods for selective gene regulation
CN111088285B (zh) * 2019-08-15 2021-12-28 北京锦篮基因科技有限公司 携带atp7b基因表达框及变异体的aav载体及应用
JP7451705B2 (ja) * 2019-11-26 2024-03-18 コリア リサーチ インスティテュート オブ バイオサイエンス アンド バイオテクノロジー タンパク質発現用ｍＲＮＡ構造体及びその用途
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
WO2022208342A1 (en) * 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
CN118434854A (zh) * 2021-10-18 2024-08-02 逻辑生物治疗公司 用于治疗威尔森氏病的基因疗法
WO2023230466A1 (en) * 2022-05-23 2023-11-30 Lutsenka Svetlana Compositions and methods for treating wilson's disease
AU2024239180A1 (en) * 2023-03-22 2025-11-06 Skyline Therapeutics Limited Recombinant aav for the gene therapy of wilson disease
US20240360431A1 (en) * 2023-04-30 2024-10-31 Sichuan Real&Best Biotech Co., Ltd. Engineered alpha-galactosidase a (a-gal a) peptides and functional variants thereof and associated methods of treating fabry disease
CN120882865A (zh) * 2023-07-28 2025-10-31 凌意(杭州)生物科技有限公司 用于治疗威尔逊病的多核苷酸
WO2025110984A1 (en) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions and methods for treating wilson's disease

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030228282A1 (en) * 2001-12-17 2003-12-11 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
WO2016097218A1 (en) * 2014-12-17 2016-06-23 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions
WO2016097219A1 (en) * 2014-12-17 2016-06-23 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease
CN113302290A (zh) * 2018-11-16 2021-08-24 编码治疗公司 治疗威尔逊氏病的组合物和方法

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2002350039A1 (en) 2001-10-29 2003-05-12 Renovis, Inc. Method for isolating cell-type specific mrnas
DE10156121A1 (de) 2001-11-16 2003-05-28 Wolfgang Stremmel Somatische Gentherapie mit Vektoren, die einen verkürzten metallsensitiven Promotor besitzen, zur Behandlung des Morbus Wilson sowie anderer Erkrankungen, die mit Veränderungen im Metallstoffwechsel einhergehen, wie z.B. Alzheimer-Erkrankung
EP1660970A4 (en) 2003-08-01 2007-02-14 Dna Twopointo Inc SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
ES2874298T3 (es) 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US20150045284A1 (en) 2011-01-21 2015-02-12 Aarhus Universitet CRYSTAL STRUCTURE OF A TYPE IB P-TYPE ATPase
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
JP6373763B2 (ja) 2012-02-17 2018-08-22 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法
AU2013243954A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
CN110290814A (zh) * 2016-12-30 2019-09-27 宾夕法尼亚州立大学托管会 用于治疗威尔逊病的基因疗法
JP7531495B2 (ja) 2019-01-04 2024-08-09 ウルトラジェニックス ファーマシューティカル インコーポレイテッド ウイルソン病を処置するための遺伝子治療構築物

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030228282A1 (en) * 2001-12-17 2003-12-11 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
WO2016097218A1 (en) * 2014-12-17 2016-06-23 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions
WO2016097219A1 (en) * 2014-12-17 2016-06-23 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease
CN113302290A (zh) * 2018-11-16 2021-08-24 编码治疗公司 治疗威尔逊氏病的组合物和方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CHUAH MK等: "Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates", 《MOL THER.》 *
OIHANA MURILLO等: "Long-term metabolic correction of Wilson"s disease in a murine model by gene therapy", 《J HEPATOL》 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115216481A (zh) * 2021-04-21 2022-10-21 四川至善唯新生物科技有限公司 一种表达量提高的截短型atp7b基因及其应用
CN115216533A (zh) * 2022-06-30 2022-10-21 湖南家辉生物技术有限公司 一种用于诊断威尔逊病的生物标志物、扩增引物组、检测试剂及应用
CN120457199A (zh) * 2023-03-06 2025-08-08 凌意(杭州)生物科技有限公司 截短的atp7b及其用途
CN117947035A (zh) * 2024-03-25 2024-04-30 上海凌医生物科技有限公司 一种增强基因表达的启动子

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US20190338310A1 (en) 2019-11-07
WO2018126116A1 (en) 2018-07-05
KR102604096B1 (ko) 2023-11-23
BR112019013245A2 (pt) 2020-02-11
EP3562514A1 (en) 2019-11-06
MX2019007873A (es) 2019-11-18
JP7128190B2 (ja) 2022-08-30
EP4361277A3 (en) 2024-07-31
US12460228B2 (en) 2025-11-04
KR20230160411A (ko) 2023-11-23
CA3048044A1 (en) 2018-07-05
EP3562514A4 (en) 2020-08-05
US20220389455A1 (en) 2022-12-08
KR102863181B1 (ko) 2025-09-23
EP4361277A2 (en) 2024-05-01
KR20190101410A (ko) 2019-08-30
JP2020514286A (ja) 2020-05-21
US11473106B2 (en) 2022-10-18

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