SI2579900T1 - Vektorji in zaporedja za zdravljenje bolezni - Google Patents
Vektorji in zaporedja za zdravljenje bolezni Download PDFInfo
- Publication number
- SI2579900T1 SI2579900T1 SI201131405T SI201131405T SI2579900T1 SI 2579900 T1 SI2579900 T1 SI 2579900T1 SI 201131405 T SI201131405 T SI 201131405T SI 201131405 T SI201131405 T SI 201131405T SI 2579900 T1 SI2579900 T1 SI 2579900T1
- Authority
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- Slovenia
- Prior art keywords
- composition according
- gene composition
- plasmid
- nucleotide sequence
- vector
- Prior art date
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- 239000013598 vector Substances 0.000 title claims abstract 11
- 201000010099 disease Diseases 0.000 title claims abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title claims abstract 3
- 108090000623 proteins and genes Proteins 0.000 claims abstract 15
- 239000008194 pharmaceutical composition Substances 0.000 claims abstract 4
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims abstract 2
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims abstract 2
- 239000000203 mixture Substances 0.000 claims 13
- 239000002773 nucleotide Substances 0.000 claims 8
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 239000013612 plasmid Substances 0.000 claims 8
- 239000013604 expression vector Substances 0.000 claims 3
- 229920002683 Glycosaminoglycan Polymers 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 238000001890 transfection Methods 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 238000010276 construction Methods 0.000 abstract 1
Classifications
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- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/67—General methods for enhancing the expression
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
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- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- 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
- 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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- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
- C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Saccharide Compounds (AREA)
Claims (15)
- VEKTORJI IN ZAPOREDJA ZA ZDRAVLJENJE BOLEZNI Patentni zahtevki1. Izolirano nukleotidno zaporedje z ID ŠT. ZAPOREDJA:*!, ki kodira protein z ID ŠT. ZAPOREDJA: 2.
- 2. Genska sestava, ki zajema nukleotidno zaporedje v skladu s patentnim zahtevkom 1.
- 3. Genska sestava v skladu s patentnim zahtevkom 2, pri čemer je omenjena genska sestava izrazni vektor.
- 4. Genska sestava v skladu s patentnim zahtevkom 3, pri čemer je vektor adenoasociirani vektor.
- 5. Genska sestava v skladu s patentnim zahtevkom 4, pri čemer je serotip adenoasociiranega vektorja 1,2, 5, 7, 8 ali 9.
- 6. Genska sestava v skladu s patentnim zahtevkom 4, ki zajema promotor CAG, operabilno vezan z ID ŠT. ZAPOREDJA: 1.
- 7. Genska sestava v skladu s patentnim zahtevkom 6, ki je adenoasociirani vektor serotipa 9 (AAV9), ki zajema promotor CAG, operabilno vezan z ID ŠT. ZAPOREDJA: 1 (co-hu-SFMD), pri čemer je omenjena genska sestava označena kot AAV9-CAG-co-hu-SFMD.
- 8. Plazmid z referenčno številko DSM 24817, ki vsebuje nukleotidno zaporedje v skladu s patentnim zahtevkom 1 (co-hu-SFMD) in promotor CAG, pri čemer je omenjeni plazmid označen kot pAAV-CAG-co-hu-SFMD.
- 9. Genska sestava v skladu s patentnim zahtevkom 3, ki zajema promotor hAAT, operabilno vezan z ID ŠT. ZAPOREDJA: 1.
- 10. Plazmid, ki vsebuje nukleotidno zaporedje v skladu s patentnim zahtevkom 1 (co-hu-SFMD), operabilno vezano s promotorjem hAAT, pri čemer je omenjeni plazmid označen kot pAAV-hAAT-co-hu-SFMD.
- 11. Farmacevtska sestava, ki zajemanukleotidno zaporedje v skladu s patentnim zahtevkom 1; gensko sestavo v skladu s katerim koli patentnim zahtevkom od 2 do 7 in 9; ali plazmid v skladu s patentnim zahtevkom 8 ali 10.
- 12. Nukleotidno zaporedje v skladu s patentnim zahtevkom 1; genska sestava v skladu s katerim koli patentnim zahtevkom od 2 do 7 in 9; ali plazmid v skladu s patentnim zahtevkom 8 ali 10; ali farmacevtska sestava v skladu s patentnim zahtevkom 11 za uporabo kot zdravilo.
- 13. Nukleotidno zaporedje v skladu s patentnim zahtevkom 1; genska sestava v skladu s katerim koli patentnim zahtevkom od 2 do 7 in 9; ali plazmid v skladu s patentnim zahtevkom 8 ali 10; ali farmacevtska sestava v skladu s patentnim zahtevkom 11 za zdravljenje mukopolisaharidoz, po možnosti mukopolisaharidoze tipa IMA ali Sanfilippovega sindroma.
- 14. Metoda za pridobivanje izraznih vektorjev v skladu s patentnim zahtevkom 4, ki zajema naslednje korake: i) zagotavljanje prvega vektorja, ki zajema ID ŠT. ZAPOREDJA: 1, vstavljenega med prvo ponovitvijo konca AAV in drugo ponovitvijo konca AAV, promotorja CAG ali hAAT, operabilno vezanega z ID ŠT. ZAPORED JA: 1; drugega vektorja, ki zajema gen rep AAV in gen cap AAV; in tretjega vektorja, ki zajema funkcijo adenovirusa pomočnika; ii) sotransfekcija ustreznih celic z vektorji iz koraka i); iii) vzgajanje transfekcijskih celic iz koraka ii); in iv) čiščenje izraznih vektorjev iz kulture iz koraka iii).
- 15. Izolirana celica, ki vsebuje: nukleotid no zaporedje v skladu s patentnim zahtevkom 1; gensko sestavo v skladu s katerim koli patentnim zahtevkom od 2 do 7 in 9; ali plazmid v skladu s patentnim zahtevkom 8 ali 10.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP20100382169 EP2394667A1 (en) | 2010-06-10 | 2010-06-10 | Vectors and sequences for the treatment of diseases |
EP11724246.1A EP2579900B1 (en) | 2010-06-10 | 2011-06-10 | Vectors and sequences for the treatment of diseases |
PCT/EP2011/059678 WO2011154520A1 (en) | 2010-06-10 | 2011-06-10 | Vectors and sequences for the treatment of diseases |
Publications (1)
Publication Number | Publication Date |
---|---|
SI2579900T1 true SI2579900T1 (sl) | 2018-04-30 |
Family
ID=42710651
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SI201131405T SI2579900T1 (sl) | 2010-06-10 | 2011-06-10 | Vektorji in zaporedja za zdravljenje bolezni |
Country Status (31)
Country | Link |
---|---|
US (2) | US8999948B2 (sl) |
EP (2) | EP2394667A1 (sl) |
JP (1) | JP6066902B2 (sl) |
KR (1) | KR101952966B1 (sl) |
CN (1) | CN103037905B (sl) |
AR (1) | AR081868A1 (sl) |
AU (1) | AU2011263697B2 (sl) |
BR (1) | BR112012031067B1 (sl) |
CA (1) | CA2801639C (sl) |
CY (1) | CY1119892T1 (sl) |
DK (1) | DK2579900T3 (sl) |
ES (2) | ES2659031T3 (sl) |
HR (1) | HRP20180146T1 (sl) |
HU (1) | HUE038190T2 (sl) |
IL (1) | IL223274A (sl) |
LT (1) | LT2579900T (sl) |
ME (1) | ME02940B (sl) |
MX (1) | MX2012014372A (sl) |
MY (1) | MY172292A (sl) |
NO (1) | NO2579900T3 (sl) |
NZ (1) | NZ603901A (sl) |
PL (1) | PL2579900T3 (sl) |
PT (1) | PT2579900T (sl) |
RS (1) | RS57458B1 (sl) |
RU (1) | RU2588667C2 (sl) |
SG (2) | SG186136A1 (sl) |
SI (1) | SI2579900T1 (sl) |
TW (1) | TWI547288B (sl) |
UA (1) | UA112841C2 (sl) |
WO (1) | WO2011154520A1 (sl) |
ZA (1) | ZA201209103B (sl) |
Families Citing this family (58)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA3128549A1 (en) | 2010-07-12 | 2012-01-19 | Universitat Autonoma De Barcelona | Gene therapy composition for use in diabetes treatment |
US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
EP2492347A1 (en) * | 2012-05-22 | 2012-08-29 | Laboratorios Del. Dr. Esteve, S.A. | Methods for the production of vectors |
EP2692868A1 (en) | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
EP3747998A1 (en) | 2013-03-15 | 2020-12-09 | The Trustees of the University of Pennsylvania | Compositions for treating mpsi |
CN115120745A (zh) * | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
KR20220106852A (ko) * | 2013-07-26 | 2022-07-29 | 유니버시티 오브 아이오와 리써치 파운데이션 | 뇌 질환을 치료하기 위한 방법 및 조성물 |
RU2708026C2 (ru) * | 2014-04-01 | 2019-12-03 | Свидиш Орфан Биовитрум Аб (Пабл) | Модифицированная сульфамидаза и способ её получения |
PT3143146T (pt) * | 2014-05-14 | 2020-08-27 | Univ Auta²Noma De Barcelona | Vetores de vírus adenoassociados para o tratamento de distúrbios de armazenamento lisossomal |
EP3194430A1 (en) * | 2014-09-16 | 2017-07-26 | Universitat Autònoma De Barcelona | Adeno-associated viral vectors for the gene therapy of metabolic diseases |
GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
ES2824457T3 (es) | 2014-12-05 | 2021-05-12 | UNIV AUTòNOMA DE BARCELONA | Vectores virales para el tratamiento de la diabetes |
EP3233131A1 (en) | 2014-12-16 | 2017-10-25 | Board of Regents of the University of Nebraska | Gene therapy for juvenile batten disease |
PL3233130T3 (pl) | 2014-12-17 | 2021-11-22 | Fundacion Para La Investigacion Medica Aplicada | Konstrukty kwasów nukleinowych i wektory do terapii genowej do zastosowania w leczeniu choroby wilsona |
JP6956635B2 (ja) * | 2014-12-17 | 2021-11-02 | フンダシオン パラ ラ インベスティガシオン メディカ アプリカダ | ウィルソン病及び他の状態の処置に使用するための核酸構築物及び遺伝子治療ベクター |
EP3978614A3 (en) | 2015-01-07 | 2022-07-27 | Universitat Autònoma de Barcelona | Single-vector gene construct comprising insulin and glucokinase genes |
MX2017011615A (es) * | 2015-03-10 | 2018-04-11 | Univ Columbia | Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1. |
US20180289839A1 (en) * | 2015-05-15 | 2018-10-11 | Regents Of The University Of Minnesota | Intranasal therapeutic delivery of adeno-associated virus to central nervous system |
EP3101125A1 (en) * | 2015-06-05 | 2016-12-07 | Laboratorios Del Dr. Esteve, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
PE20181156A1 (es) * | 2015-11-05 | 2018-07-19 | Bamboo Therapeutics Inc | Genes de ataxia de friedreich modificados y vectores para terapia genica |
SG11201806270XA (en) | 2016-02-03 | 2018-08-30 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
IL305449A (en) | 2016-04-15 | 2023-10-01 | Univ Pennsylvania | Gene therapy for the treatment of type II mucositis |
CA3019425A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia a |
EP3485005A1 (en) * | 2016-07-12 | 2019-05-22 | The University Of Manchester | Gene therapy |
BR112019001887A2 (pt) | 2016-08-02 | 2019-07-09 | Editas Medicine Inc | composições e métodos para o tratamento de doença associada a cep290 |
AU2017313844B2 (en) | 2016-08-19 | 2023-10-12 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus |
CN109863243B (zh) * | 2016-09-30 | 2023-07-28 | 埃斯蒂文制药股份有限公司 | 用于治疗粘多糖症的腺相关病毒载体 |
US11958886B2 (en) | 2016-12-07 | 2024-04-16 | University Of Florida Research Foundation, Incorporated | IL-1RA cDNAs |
GB201707212D0 (en) * | 2017-05-05 | 2017-06-21 | Ucl Business Plc | Gene therapy for ciliopathies |
EP3622060A4 (en) * | 2017-05-12 | 2021-02-17 | The Children's Hospital of Philadelphia | SULFAMIDASE (SGSH) VARIANTS, VECTORS, COMPOSITIONS AND METHODS AND USES FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE IIIA (MPS IIIA) |
BR112020000063A2 (pt) | 2017-07-06 | 2020-07-14 | The Trustees Of The University Of Pennsylvania | terapia gênica mediada por aav9 para tratamento de mucopolissacaridose tipo i |
WO2019028306A2 (en) | 2017-08-03 | 2019-02-07 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES |
CA3076036A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
CA3083416A1 (en) * | 2017-11-30 | 2019-06-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis iii a |
EP3717652A4 (en) * | 2017-11-30 | 2021-11-24 | The Trustees of the University of Pennsylvania | GENE THERAPY FOR MUCOPOLY SACCHARIDOSIS IIIB |
US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
WO2019161365A1 (en) | 2018-02-19 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof |
SG11202011296VA (en) | 2018-05-15 | 2020-12-30 | Voyager Therapeutics Inc | Compositions and methods for the treatment of parkinson's disease |
TW202015742A (zh) | 2018-05-15 | 2020-05-01 | 美商航海家醫療公司 | 投遞腺相關病毒(aav)之組成物和方法 |
EP3793615A2 (en) | 2018-05-16 | 2021-03-24 | Voyager Therapeutics, Inc. | Directed evolution of aav to improve tropism for cns |
CN108728495A (zh) * | 2018-05-31 | 2018-11-02 | 深圳市免疫基因治疗研究院 | 一种Sanfilippo A综合症慢病毒载体、慢病毒及其制备方法和应用 |
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
WO2020127678A1 (en) * | 2018-12-20 | 2020-06-25 | Esteve Pharmaceuticals, S.A. | Recombinant vectors for the long term treatment of mucchopolysacharidosis |
EP3911410A1 (en) | 2019-01-18 | 2021-11-24 | Voyager Therapeutics, Inc. | Methods and systems for producing aav particles |
TW202039856A (zh) * | 2019-03-28 | 2020-11-01 | 西班牙商艾斯提夫製藥股份有限公司 | 製造重組病毒載體之方法 |
US20220333133A1 (en) | 2019-09-03 | 2022-10-20 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
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 |
CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神(北京)生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
MX2023000815A (es) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de trastornos neurologicos relacionados con la deficiencia de glucosilceramidasa beta. |
WO2022026410A2 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
EP4229192A1 (en) | 2020-10-14 | 2023-08-23 | Denali Therapeutics Inc. | Fusion proteins comprising sulfoglucosamine sulfohydrolase enzymes and methods thereof |
WO2023044483A2 (en) | 2021-09-20 | 2023-03-23 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
WO2023091949A2 (en) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
WO2023150051A1 (en) * | 2022-02-04 | 2023-08-10 | M6P Therapeutics, Inc. | Compositions and methods of using two-promoter vector for treatment of lysosomal storage disorders |
CN115029360A (zh) * | 2022-05-30 | 2022-09-09 | 上海勉亦生物科技有限公司 | 用于治疗粘多糖贮积症iiia型的转基因表达盒 |
WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
Family Cites Families (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO1999002694A1 (en) * | 1997-07-09 | 1999-01-21 | The University Of Queensland | Nucleic acid sequence and method for selectively expressing a protein in a target cell or tissue |
GB0100889D0 (en) * | 2001-01-12 | 2001-02-21 | Oxford Glycosciences Uk Ltd | Compounds |
WO2001036603A2 (en) * | 1999-11-17 | 2001-05-25 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
US7351813B2 (en) * | 2000-06-20 | 2008-04-01 | The Board Of Trustees Of The Leland Stanford Junior University | Liver-specific gene expression cassettes, and methods of use |
EP1521523A4 (en) * | 2002-05-20 | 2006-04-19 | Univ Texas | METHODS AND COMPOSITIONS FOR DELIVERING NUCLEIC ACID ENZYMES AND MOLECULES IN BRAIN, BONE, AND OTHER FABRICS |
WO2004042059A1 (en) * | 2002-11-08 | 2004-05-21 | The University Of Queensland | A method for optimising gene expression using synonymous codon optimisation |
WO2004072275A2 (en) * | 2003-02-11 | 2004-08-26 | Transkaryotic Therapies, Inc. | Diagnosis and treatment of multiple sulfatase deficiency and other using a formylglycine generating enzyme (fge) |
WO2008085912A1 (en) | 2007-01-05 | 2008-07-17 | Massachusetts Institute Of Technology | Compositions of and methods of using sulfatases from flavobacterium heparinum |
DK2191001T3 (en) * | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
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