CY1119295T1 - Γονιδιακη θεραπεια για ασθενειες λυσοσωμικης αποθhκευσης - Google Patents
Γονιδιακη θεραπεια για ασθενειες λυσοσωμικης αποθhκευσηςInfo
- Publication number
- CY1119295T1 CY1119295T1 CY20171100836T CY171100836T CY1119295T1 CY 1119295 T1 CY1119295 T1 CY 1119295T1 CY 20171100836 T CY20171100836 T CY 20171100836T CY 171100836 T CY171100836 T CY 171100836T CY 1119295 T1 CY1119295 T1 CY 1119295T1
- Authority
- CY
- Cyprus
- Prior art keywords
- subject
- transgene
- brain
- storage diseases
- lysosomic
- Prior art date
Links
Classifications
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
-
- 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
- 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
Abstract
Αυτή η περιγραφή παρέχει μεθόδους και συνθέσεις για την θεραπεία ασθενειών λυσοσωμικής αποθήκευσης σε ένα υποκείμενο. Σε μία άποψη της εφεύρεσης, ένα διαγονιδιακό προϊόν χορηγείται σε ένα υποκείμενο με την χορήγηση ενός ανασυνδυασμένου νευροτροφικού ιικού φορέα που περιέχει το διαγονίδιο στον εγκέφαλο. Ο ιικός φορέας παραδίδει το διαγονίδιο σε μια περιοχή του εγκεφάλου που είναι ευάλωτη σε μόλυνση από τον ιό και η οποία εκφράζει το κωδικοποιημένο προϊόν ανασυνδυασμένου ιικού γονιδίου. Παρέχονται επίσης συνθέσεις για την παράδοση ενός διαγονιδιακού προϊόντος σε ένα υποκείμενο με την χορήγηση ενός ανασυνδυασμένου νευροτροφικού ιικού φορέα που περιέχει το διαγονίδιο στον εγκέφαλο του υποκειμένου. Το διαγονιδιακό προϊόν μπορεί να είναι οποιοδήποτε που είναι ανεπαρκές σε μία ασθένεια λυσοσωμικής αποθήκευσης.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US92494707P | 2007-06-06 | 2007-06-06 | |
PCT/US2008/065481 WO2008154198A1 (en) | 2007-06-06 | 2008-06-02 | Gene therapy for lysosomal storage diseases |
Publications (1)
Publication Number | Publication Date |
---|---|
CY1119295T1 true CY1119295T1 (el) | 2018-02-14 |
Family
ID=40130114
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20171100836T CY1119295T1 (el) | 2007-06-06 | 2017-08-03 | Γονιδιακη θεραπεια για ασθενειες λυσοσωμικης αποθhκευσης |
Country Status (14)
Country | Link |
---|---|
US (2) | US8796236B2 (el) |
EP (2) | EP3252161B1 (el) |
AR (1) | AR070000A1 (el) |
CY (1) | CY1119295T1 (el) |
DK (1) | DK2158322T3 (el) |
ES (2) | ES2635726T3 (el) |
HR (1) | HRP20171165T1 (el) |
HU (1) | HUE035779T2 (el) |
IL (3) | IL202522A (el) |
LT (1) | LT2158322T (el) |
PL (2) | PL3252161T3 (el) |
PT (2) | PT2158322T (el) |
SI (1) | SI2158322T1 (el) |
WO (1) | WO2008154198A1 (el) |
Families Citing this family (31)
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AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
EP1988823B1 (en) | 2006-02-09 | 2018-08-15 | Genzyme Corporation | Slow intraventricular delivery |
ES2635726T3 (es) | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US8242086B2 (en) * | 2008-11-12 | 2012-08-14 | Duke University | Methods and compositions for treating disorders caused by a deficiency in a gene product of a CLN gene |
ES2786039T3 (es) | 2009-04-30 | 2020-10-08 | Ospedale San Raffaele Srl | Vector génico |
US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
DK2826860T3 (en) | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
US9849195B2 (en) | 2011-03-31 | 2017-12-26 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
US20130090374A1 (en) * | 2011-04-20 | 2013-04-11 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis |
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 |
LT2751279T (lt) | 2011-08-31 | 2017-12-11 | St. Jude Children`S Research Hospital | Būdai ir kompozicijos lizosominės egzocitozės aktyvumo lygio nustatymui ir panaudojimo būdai |
CN115120746A (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 |
US11078464B2 (en) | 2013-08-30 | 2021-08-03 | Amgen Inc. | High titer recombinant AAV vector production in adherent and suspension cells |
CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
MX2017005834A (es) | 2014-11-05 | 2017-11-17 | Voyager Therapeutics Inc | Polinucleotidos aad para el tratamiento de la enfermedad de parkinson. |
CA3019315A1 (en) * | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
RU2742612C2 (ru) * | 2015-12-15 | 2021-02-09 | Джензим Корпорейшн | Векторы на основе аденоассоциированного вируса для лечения муколипидоза типа ii |
AU2017209189B2 (en) * | 2016-01-19 | 2024-03-28 | The Regents Of The University Of California | Methods for the treatment of Danon disease and other disorders of autophagy |
JP7171439B2 (ja) | 2016-04-15 | 2022-11-15 | 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 | ムコ多糖症ii型を処置するための遺伝子療法 |
US11578340B2 (en) | 2016-10-13 | 2023-02-14 | University Of Massachusetts | AAV capsid designs |
CA3039673A1 (en) | 2016-10-20 | 2018-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
WO2018085688A1 (en) * | 2016-11-04 | 2018-05-11 | The Children's Hospital Of Philadelphia | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
US11819539B2 (en) | 2017-09-22 | 2023-11-21 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating Mucopolysaccharidosis type II |
EP3794131A4 (en) * | 2018-05-15 | 2022-01-26 | University of Massachusetts | RECOMBINANT AAV VECTORS CODING FOR LYSOSOMIAL BETA-GALACTOSIDASE (GLB1) AND CATHEPSIN A |
WO2020014523A1 (en) | 2018-07-12 | 2020-01-16 | Rocket Pharmaceuticals, Ltd. | Gene therapy vectors for treatment of danon disease |
KR20220007601A (ko) * | 2019-04-12 | 2022-01-18 | 엔코디드 테라퓨틱스, 인크. | 치료제 투여를 위한 조성물 및 방법 |
CN115379863A (zh) * | 2020-04-14 | 2022-11-22 | 吉尼松公司 | 用于治疗酸性神经酰胺酶缺乏症的载体 |
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US5672344A (en) | 1987-12-30 | 1997-09-30 | The Regents Of The University Of Michigan | Viral-mediated gene transfer system |
US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
JP3532566B2 (ja) | 1993-06-24 | 2004-05-31 | エル. グラハム,フランク | 遺伝子治療のためのアデノウイルスベクター |
ATE437232T1 (de) | 1993-10-25 | 2009-08-15 | Canji Inc | Rekombinanter adenoviren-vektor und verfahren zur verwendung |
DE69433592T2 (de) | 1993-11-09 | 2005-02-10 | Targeted Genetics Corp., Seattle | Die erzielung hoher titer des rekombinanten aav-vektors |
US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
CA2187626C (en) | 1994-04-13 | 2009-11-03 | Michael G. Kaplitt | Aav-mediated delivery of dna to cells of the nervous system |
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US20050026832A1 (en) * | 1997-11-25 | 2005-02-03 | Genentech, Inc. | Fibroblast growth factor-19 (FGF-19) nucleic acids and polypeptides and methods of use for the treatment of obesity and related disorders |
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US6730297B1 (en) * | 1999-05-28 | 2004-05-04 | Chiron Corporation | Use of recombinant gene delivery vectors for treating or preventing lysosomal storage disorders |
WO2001036603A2 (en) * | 1999-11-17 | 2001-05-25 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
AU2001236713A1 (en) * | 2000-02-17 | 2001-08-27 | Genzyme Corporation | Methods for treatment of lysosomal storage diseases |
US20040204379A1 (en) * | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
EP1319082B1 (en) | 2000-09-18 | 2005-11-16 | Genzyme Corporation | Expression vectors containing hybrid ubiquitin promoters |
US20040258666A1 (en) | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
CN101027388A (zh) | 2003-06-12 | 2007-08-29 | 建新公司 | 具有增强活性的修饰的人酸性神经磷脂酶及其制备方法 |
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US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
US20060171926A1 (en) * | 2004-04-30 | 2006-08-03 | Passini Marco A | Gene therapy for neurometabolic disorders |
ATE525092T1 (de) * | 2005-05-02 | 2011-10-15 | Genzyme Corp | Gentherapie für neurometabolische erkrankungen |
ES2700048T3 (es) * | 2006-02-08 | 2019-02-13 | Genzyme Corp | Terapia génica par la enfermedad de Niemann-Pick tipo A |
EP1988823B1 (en) * | 2006-02-09 | 2018-08-15 | Genzyme Corporation | Slow intraventricular delivery |
ES2635726T3 (es) | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
EP3482767B1 (en) | 2009-08-28 | 2021-10-06 | Icahn School of Medicine at Mount Sinai | Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency |
-
2008
- 2008-06-02 ES ES08756596.6T patent/ES2635726T3/es active Active
- 2008-06-02 EP EP17168189.3A patent/EP3252161B1/en active Active
- 2008-06-02 WO PCT/US2008/065481 patent/WO2008154198A1/en active Application Filing
- 2008-06-02 DK DK08756596.6T patent/DK2158322T3/en active
- 2008-06-02 PT PT87565966T patent/PT2158322T/pt unknown
- 2008-06-02 SI SI200831847T patent/SI2158322T1/sl unknown
- 2008-06-02 PL PL17168189T patent/PL3252161T3/pl unknown
- 2008-06-02 PL PL08756596T patent/PL2158322T3/pl unknown
- 2008-06-02 EP EP08756596.6A patent/EP2158322B1/en active Active
- 2008-06-02 LT LTEP08756596.6T patent/LT2158322T/lt unknown
- 2008-06-02 ES ES17168189T patent/ES2905616T3/es active Active
- 2008-06-02 HU HUE08756596A patent/HUE035779T2/en unknown
- 2008-06-02 PT PT171681893T patent/PT3252161T/pt unknown
- 2008-06-06 AR ARP080102421A patent/AR070000A1/es unknown
-
2009
- 2009-12-04 US US12/631,336 patent/US8796236B2/en active Active
- 2009-12-06 IL IL202522A patent/IL202522A/en active IP Right Grant
-
2014
- 2014-07-10 US US14/328,621 patent/US11369693B2/en active Active
-
2017
- 2017-06-14 IL IL252898A patent/IL252898B/en active IP Right Grant
- 2017-07-31 HR HRP20171165TT patent/HRP20171165T1/hr unknown
- 2017-08-03 CY CY20171100836T patent/CY1119295T1/el unknown
-
2019
- 2019-05-19 IL IL266734A patent/IL266734B/en unknown
Also Published As
Publication number | Publication date |
---|---|
PT3252161T (pt) | 2022-02-01 |
IL202522A (en) | 2017-10-31 |
PL3252161T3 (pl) | 2022-03-07 |
HUE035779T2 (en) | 2018-05-28 |
PT2158322T (pt) | 2017-08-09 |
US8796236B2 (en) | 2014-08-05 |
EP3252161A1 (en) | 2017-12-06 |
IL266734A (en) | 2019-07-31 |
EP2158322A4 (en) | 2011-06-01 |
WO2008154198A1 (en) | 2008-12-18 |
IL266734B (en) | 2021-10-31 |
SI2158322T1 (sl) | 2017-10-30 |
HRP20171165T1 (hr) | 2017-12-15 |
ES2905616T3 (es) | 2022-04-11 |
US20100173979A1 (en) | 2010-07-08 |
EP2158322B1 (en) | 2017-05-03 |
LT2158322T (lt) | 2017-08-25 |
IL202522A0 (en) | 2011-08-01 |
DK2158322T3 (en) | 2017-08-28 |
ES2635726T3 (es) | 2017-10-04 |
PL2158322T3 (pl) | 2017-10-31 |
AR070000A1 (es) | 2010-03-10 |
IL252898B (en) | 2019-06-30 |
US20150151007A1 (en) | 2015-06-04 |
EP3252161B1 (en) | 2021-11-17 |
EP2158322A1 (en) | 2010-03-03 |
IL252898A0 (en) | 2017-08-31 |
US11369693B2 (en) | 2022-06-28 |
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