ATE521701T1 - Verbesserte konstrukte zur expression lysosomaler polypeptide - Google Patents
Verbesserte konstrukte zur expression lysosomaler polypeptideInfo
- Publication number
- ATE521701T1 ATE521701T1 AT04704039T AT04704039T ATE521701T1 AT E521701 T1 ATE521701 T1 AT E521701T1 AT 04704039 T AT04704039 T AT 04704039T AT 04704039 T AT04704039 T AT 04704039T AT E521701 T1 ATE521701 T1 AT E521701T1
- Authority
- AT
- Austria
- Prior art keywords
- gaa
- lysosomal
- utr
- isolated nucleic
- polypeptide
- Prior art date
Links
- 229920001184 polypeptide Polymers 0.000 title abstract 7
- 108090000765 processed proteins & peptides Proteins 0.000 title abstract 7
- 102000004196 processed proteins & peptides Human genes 0.000 title abstract 7
- 230000002132 lysosomal effect Effects 0.000 title abstract 6
- 108020004707 nucleic acids Proteins 0.000 abstract 4
- 102000039446 nucleic acids Human genes 0.000 abstract 4
- 150000007523 nucleic acids Chemical class 0.000 abstract 4
- 108020005345 3' Untranslated Regions Proteins 0.000 abstract 3
- 108091026890 Coding region Proteins 0.000 abstract 3
- 239000013598 vector Substances 0.000 abstract 2
- 101710096786 Lysosomal acid alpha-glucosidase Proteins 0.000 abstract 1
- 108010076504 Protein Sorting Signals Proteins 0.000 abstract 1
- 108091036066 Three prime untranslated region Proteins 0.000 abstract 1
- 108091023045 Untranslated Region Proteins 0.000 abstract 1
- 230000007812 deficiency Effects 0.000 abstract 1
- 238000012217 deletion Methods 0.000 abstract 1
- 230000037430 deletion Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 230000003248 secreting effect Effects 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/036—Fusion polypeptide containing a localisation/targetting motif targeting to the medium outside of the cell, e.g. type III secretion
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Chemical & Material Sciences (AREA)
- Obesity (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Veterinary Medicine (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US44178903P | 2003-01-22 | 2003-01-22 | |
| PCT/US2004/001453 WO2004064750A2 (en) | 2003-01-22 | 2004-01-21 | Improved constructs for expressing lysosomal polypeptides |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ATE521701T1 true ATE521701T1 (de) | 2011-09-15 |
Family
ID=32771976
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AT04704039T ATE521701T1 (de) | 2003-01-22 | 2004-01-21 | Verbesserte konstrukte zur expression lysosomaler polypeptide |
Country Status (7)
| Country | Link |
|---|---|
| US (3) | US20040248262A1 (de) |
| EP (1) | EP1587923B1 (de) |
| JP (1) | JP5433133B2 (de) |
| AT (1) | ATE521701T1 (de) |
| ES (1) | ES2371913T3 (de) |
| PT (1) | PT1587923E (de) |
| WO (1) | WO2004064750A2 (de) |
Families Citing this family (62)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2002256423B2 (en) * | 2001-04-30 | 2008-07-24 | Zystor Therapeutics, Inc. | Subcellular targeting of therapeutic proteins |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US7629309B2 (en) * | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| JP4914224B2 (ja) * | 2004-02-10 | 2012-04-11 | バイオマリン ファーマシューティカル インコーポレイテッド | 酸性αグルコシダーゼおよびそのフラグメント |
| US8889127B2 (en) * | 2004-07-01 | 2014-11-18 | Icahn School Of Medicine At Mount Sinai | Targeted protein replacement for the treatment of lysosomal storage disorders |
| KR20080038131A (ko) * | 2005-07-18 | 2008-05-02 | 프로탈릭스 리미티드 | 생물학적으로 활성인 거대분자의 점막 또는 장관 투여 |
| FR2889066B1 (fr) * | 2005-07-28 | 2007-11-09 | Centre Nat Rech Scient | Procede d'immunisation genetique par electrotransfert contre une toxine et antiserum susceptible d'etre obtenu par ledit procede |
| EP2099523A2 (de) * | 2006-11-13 | 2009-09-16 | ZyStor Therapeutics , Inc. | Verfahren zur behandlung von pompe-krankheit |
| US20120322861A1 (en) * | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| WO2009075815A1 (en) | 2007-12-07 | 2009-06-18 | Duke University | Immunomodulating gene therapy |
| US20110223147A1 (en) | 2008-05-07 | 2011-09-15 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| US20110318322A1 (en) | 2009-01-12 | 2011-12-29 | Nektar Therapeutics | Conjugates of a Lysosomal Enzyme Moiety and a Water Soluble Polymer |
| EP2215980B1 (de) * | 2009-02-04 | 2012-12-19 | Stryker Leibinger GmbH & Co. KG | Chirurgisches Elektrowerkzeug und Betätigungsbaugruppe hierfür |
| ES2405550T3 (es) * | 2009-02-04 | 2013-05-31 | Stryker Leibinger Gmbh & Co. Kg | Herramienta eléctrica quirúrgica, procedimiento de funcionamiento y grupo constructivo de accionamiento correspondientes |
| ES2758827T3 (es) | 2009-06-17 | 2020-05-06 | Biomarin Pharm Inc | Formulaciones para enzimas lisosómicas |
| BR112012033216B1 (pt) | 2010-06-25 | 2022-10-11 | Shire Human Genetic Therapies, Inc | Uso de uma formulação estável compreendendo uma proteína arilsulfatase a (asa) e formulação estável para administração intratecal |
| UA115648C2 (uk) | 2010-06-25 | 2017-12-11 | Шае Хюмен Дженетік Терапіс, Інк. | Доставка терапевтичних агентів до цнс |
| CN104857504A (zh) | 2010-06-25 | 2015-08-26 | 夏尔人类遗传性治疗公司 | 芳基硫酸酯酶a cns递送的方法和组合物 |
| PL2593131T3 (pl) | 2010-06-25 | 2020-01-31 | Shire Human Genetic Therapies, Inc. | Sposoby i kompozycje do dostarczania sulfatazy-2-iduronianiu do oun |
| RU2012154576A (ru) | 2010-06-25 | 2014-07-27 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки к цнс гепаран-n-сульфатазы |
| NZ702801A (en) | 2010-06-25 | 2016-08-26 | Shire Human Genetic Therapies | Treatment of sanfilippo syndrome type b |
| WO2012085622A1 (en) * | 2010-12-22 | 2012-06-28 | Fondazione Telethon | Therapeutic strategies to treat cns pathology in mucopolysaccharidoses |
| EP2699676A1 (de) * | 2011-04-22 | 2014-02-26 | Genzyme Corporation | Modifizierte saure alpha-glukosidase mit beschleunigter verarbeitung |
| EA201892619A1 (ru) | 2011-04-29 | 2019-04-30 | Роше Гликарт Аг | Иммуноконъюгаты, содержащие мутантные полипептиды интерлейкина-2 |
| DK2753346T3 (da) | 2011-09-07 | 2020-05-25 | Sinai School Medicine | Ceramidase og celledifferentiering |
| AU2012322811A1 (en) * | 2011-10-12 | 2014-05-01 | Synageva Biopharma Corp. | Recombinant human NaGlu protein and uses thereof |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| EP2854910B1 (de) | 2012-06-01 | 2020-04-15 | Icahn School of Medicine at Mount Sinai | Ceramidspiegel bei der behandlung und vorbeugung von infektionen |
| HUE051632T2 (hu) | 2012-06-19 | 2021-03-29 | Univ Florida | Betegségek kezelésére szolgáló készítmények és eljárások |
| US10017581B2 (en) | 2013-02-20 | 2018-07-10 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
| EP3659619A1 (de) | 2013-03-14 | 2020-06-03 | Icahn School of Medicine at Mount Sinai | Therapeutische säure-ceramidase-zusammensetzungen und verfahren zur herstellung und verwendung davon |
| US10172924B2 (en) | 2015-03-19 | 2019-01-08 | Translate Bio, Inc. | MRNA therapy for pompe disease |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| IL299325B2 (en) | 2016-09-12 | 2025-08-01 | Inst Nat Sante Rech Med | Acid-alpha glucosidase variants and uses thereof |
| EP3293260A1 (de) | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
| EP3293259A1 (de) | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
| EP3293203A1 (de) | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
| US11160493B2 (en) * | 2017-03-03 | 2021-11-02 | Musclesound, Inc. | System and method for determining a subject's muscle fuel level, muscle fuel rating, and muscle energy status |
| EP3635009B1 (de) | 2017-06-07 | 2026-02-25 | Regeneron Pharmaceuticals, Inc. | Zusammensetzungen und verfahren zur internalisierung von enzymen |
| IL276464B2 (en) | 2018-02-07 | 2026-01-01 | Regeneron Pharma | Methods and compositions for administering therapeutic protein |
| BR112020021962A2 (pt) | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| MA52626A (fr) | 2018-05-17 | 2021-03-24 | Regeneron Pharma | Anticorps anti-cd63, conjugués et leurs utilisations |
| WO2020077114A2 (en) | 2018-10-10 | 2020-04-16 | Amicus Therapeutics, Inc. | Disulfide bond stabilized polypeptide compositions and methods of use |
| CN113677800A (zh) * | 2018-11-13 | 2021-11-19 | 索元生物医药(美国)有限公司 | 包含用于结合结构域和可分泌肽的基因的重组载体 |
| AU2019381803A1 (en) * | 2018-11-16 | 2021-06-10 | AskBio Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| KR20220004696A (ko) * | 2019-04-30 | 2022-01-11 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 폼페병의 치료에 유용한 조성물 |
| US20220307055A1 (en) * | 2019-08-28 | 2022-09-29 | University Of Florida Research Foundation, Incorporated | Improved production of recombinant aav using embryonated avian eggs |
| EP4048786A1 (de) * | 2019-10-22 | 2022-08-31 | Genethon | Antikörperpolypeptide und verwendungen dafür |
| WO2021078834A1 (en) | 2019-10-22 | 2021-04-29 | Genethon | Chimeric acid-alpha glucosidase polypeptides and uses thereof |
| KR20220098384A (ko) * | 2019-11-19 | 2022-07-12 | 아스클레피오스 바이오파마슈티컬, 인크. | 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스 |
| WO2021127457A1 (en) | 2019-12-20 | 2021-06-24 | Codexis, Inc. | Engineered acid alpha-glucosidase variants |
| EP4196232A2 (de) * | 2020-08-13 | 2023-06-21 | Epivax, Inc. | Regulatorische t-zellen-epitope |
| US20240123040A1 (en) * | 2021-02-05 | 2024-04-18 | Sigilon Therapeutics, Inc. | Compositions, devices and methods for treating mps vi disease |
| AU2022379625A1 (en) * | 2021-10-27 | 2024-05-16 | The University Of North Carolina At Chapel Hill | Aav-idua vector for treatment of mps i |
| KR20250005569A (ko) * | 2022-03-18 | 2025-01-09 | 아스클레피오스 바이오파마슈티컬, 인크. | 신호 펩타이드 변형을 갖는, 폼페병을 치료하기 위한 코돈 최적화된 알파-글루코시다제(gaa) 암호화 핵산을 사용하는 치료적 아데노-연관 바이러스 |
| TW202426653A (zh) * | 2022-12-21 | 2024-07-01 | 國立臺灣大學 | 重組病毒載體、包含該重組病毒載體之重組腺相關病毒及其於治療涎酸酵素缺乏症之用途 |
| WO2025072816A2 (en) * | 2023-09-29 | 2025-04-03 | The Board Of Regents Of The University Of Texas System | Endogenous nucleus targeting signal peptides and uses thereof |
| WO2025080721A2 (en) * | 2023-10-09 | 2025-04-17 | The Board Of Regents Of The University Of Texas System | Endogenous cell membrane targeting signal peptides and uses thereof |
| WO2025097038A1 (en) * | 2023-11-03 | 2025-05-08 | The Board Of Regents Of The University Of Texas System | Endogenous cell junction targeting signal peptides and uses thereof |
| WO2025106882A1 (en) * | 2023-11-15 | 2025-05-22 | The Board Of Regents Of The University Of Texas System | Endogenous cytoplasm targeting signal peptides and uses thereof |
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| DE3023787A1 (de) | 1980-06-25 | 1982-01-21 | Studiengesellschaft Kohle mbH, 4330 Mülheim | Verfahren zur erhoehung der inkorporation und der expression von genetischem material in die kerne von intakten zellen mit hilfe von liposomen |
| US4407956A (en) | 1981-03-13 | 1983-10-04 | The Regents Of The University Of California | Cloned cauliflower mosaic virus DNA as a plant vehicle |
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| DE3432718C1 (de) | 1984-09-06 | 1986-05-22 | Biotest Pharma GmbH, 6000 Frankfurt | Verfahren zur Herstellung einer Loesung von Milch- und/oder Kolostralimmunglobulinen |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| JP2627899B2 (ja) | 1987-08-19 | 1997-07-09 | 株式会社 ビタミン研究所 | 遺伝子封入リポソームの製法 |
| FR2638643B1 (fr) * | 1988-11-09 | 1991-04-12 | Transgene Sa | Sequence d'adn codant pour le facteur ix humain ou une proteine analogue, vecteur d'expression, cellules transformees, procede de preparation du facteur ix et produits obtenus correspondants |
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-
2004
- 2004-01-21 EP EP04704039A patent/EP1587923B1/de not_active Expired - Lifetime
- 2004-01-21 ES ES04704039T patent/ES2371913T3/es not_active Expired - Lifetime
- 2004-01-21 JP JP2006501055A patent/JP5433133B2/ja not_active Expired - Lifetime
- 2004-01-21 PT PT04704039T patent/PT1587923E/pt unknown
- 2004-01-21 WO PCT/US2004/001453 patent/WO2004064750A2/en not_active Ceased
- 2004-01-21 US US10/761,530 patent/US20040248262A1/en not_active Abandoned
- 2004-01-21 AT AT04704039T patent/ATE521701T1/de not_active IP Right Cessation
-
2012
- 2012-02-23 US US13/403,783 patent/US9873868B2/en not_active Expired - Fee Related
-
2018
- 2018-01-22 US US15/877,386 patent/US20180371440A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| US9873868B2 (en) | 2018-01-23 |
| JP5433133B2 (ja) | 2014-03-05 |
| US20040248262A1 (en) | 2004-12-09 |
| WO2004064750A3 (en) | 2004-11-11 |
| EP1587923B1 (de) | 2011-08-24 |
| PT1587923E (pt) | 2011-12-07 |
| US20180371440A1 (en) | 2018-12-27 |
| ES2371913T3 (es) | 2012-01-11 |
| WO2004064750A2 (en) | 2004-08-05 |
| US20120276072A1 (en) | 2012-11-01 |
| EP1587923A2 (de) | 2005-10-26 |
| JP2006517404A (ja) | 2006-07-27 |
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