CO6541559A2 - Constructor de vector vírico novedoso para síntesis dopa continua optimizada específica de neuronas in vivo - Google Patents
Constructor de vector vírico novedoso para síntesis dopa continua optimizada específica de neuronas in vivoInfo
- Publication number
- CO6541559A2 CO6541559A2 CO12076254A CO12076254A CO6541559A2 CO 6541559 A2 CO6541559 A2 CO 6541559A2 CO 12076254 A CO12076254 A CO 12076254A CO 12076254 A CO12076254 A CO 12076254A CO 6541559 A2 CO6541559 A2 CO 6541559A2
- Authority
- CO
- Colombia
- Prior art keywords
- vector
- optimized
- continuous
- novedous
- builder
- Prior art date
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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
- C12N15/63—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
- C12N15/861—Adenoviral vectors
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
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- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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- 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
- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
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- 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
- A61P25/24—Antidepressants
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- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- 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
- A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
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- 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
- A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
- A61P25/32—Alcohol-abuse
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- 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
- A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
- A61P25/36—Opioid-abuse
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/12—Antihypertensives
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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
- C12N15/63—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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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/88—Lyases (4.)
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- 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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- C12N2750/00011—Details
- C12N2750/00041—Use of virus, viral particle or viral elements as a vector
- C12N2750/00043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- 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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- 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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- C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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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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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/60—Vector systems having a special element relevant for transcription from viruses
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- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
- C12Y114/16002—Tyrosine 3-monooxygenase (1.14.16.2)
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- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04016—GTP cyclohydrolase I (3.5.4.16)
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- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
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- Pain & Pain Management (AREA)
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Abstract
La presente invención se refiere a unsistema de expresión de un vector que comprende unasecuencia que codifica dos polipéptidos, tales comotirosina hidroxilasa (TH) y GTP-ciclohidrolasa 1 (GCH1).Los dos polipéptidos deberían ser preferencialmenteexpresados en una relación entre 3:1 y 15:1, tal como entre3:1 y 7:1. La invención es útil en el tratamiento detrastornos deficientes de catecolamina, tales comotrastornos deficientes de dopamina incluyendo pero nolimitados a mal de Parkinson. Por lo tanto, la presenteinvención proporciona un método para suministrar elconstructo de vector con objeto de limitar la producciónincrementada de la catecolamina a las células quenecesitan del mismo.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US25950209P | 2009-11-09 | 2009-11-09 |
Publications (1)
Publication Number | Publication Date |
---|---|
CO6541559A2 true CO6541559A2 (es) | 2012-10-16 |
Family
ID=43466711
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CO12076254A CO6541559A2 (es) | 2009-11-09 | 2012-05-09 | Constructor de vector vírico novedoso para síntesis dopa continua optimizada específica de neuronas in vivo |
Country Status (15)
Country | Link |
---|---|
US (2) | US20120309816A1 (es) |
EP (1) | EP2498825B1 (es) |
JP (1) | JP5894535B2 (es) |
KR (1) | KR20120089743A (es) |
CN (1) | CN102695526A (es) |
AU (1) | AU2010316982B2 (es) |
BR (1) | BR112012010866A2 (es) |
CA (1) | CA2779437A1 (es) |
CL (1) | CL2012001215A1 (es) |
CO (1) | CO6541559A2 (es) |
IL (1) | IL219704A0 (es) |
MX (1) | MX2012005450A (es) |
NZ (1) | NZ600104A (es) |
RU (1) | RU2606012C2 (es) |
WO (1) | WO2011054976A2 (es) |
Families Citing this family (56)
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US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
ES2605305T3 (es) | 2010-04-23 | 2017-03-13 | University Of Massachusetts | Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos |
CA3050894C (en) | 2010-04-23 | 2022-10-18 | University Of Massachusetts | Multicistronic expression constructs |
WO2012135389A2 (en) * | 2011-03-28 | 2012-10-04 | The Regents Of The University Of California | Host cells and methods for oxidizing aromatic amino acids |
EP3318634A1 (en) | 2011-04-21 | 2018-05-09 | University of Massachusetts | Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations |
WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
EP3750907A3 (en) | 2014-03-18 | 2021-04-28 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
WO2016054557A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Novel high efficiency library-identified aav vectors |
JP7023108B2 (ja) | 2014-10-21 | 2022-02-21 | ユニバーシティ オブ マサチューセッツ | 組み換えaavバリアントおよびその使用 |
SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
MX2017006217A (es) | 2014-11-14 | 2018-05-02 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
CN107109407A (zh) | 2014-11-14 | 2017-08-29 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
US10584321B2 (en) | 2015-02-13 | 2020-03-10 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
EP3285788A4 (en) | 2015-04-23 | 2018-12-05 | University of Massachusetts | Modulation of aav vector transgene expression |
CA3021949C (en) | 2015-04-24 | 2023-10-17 | University Of Massachusetts | Modified aav constructs and uses thereof |
MX2017014966A (es) * | 2015-05-28 | 2018-08-15 | Univ Cornell | Liberación de c1ei mediada por virus adenoasociado como terapia para el angioedema. |
KR20180034467A (ko) * | 2015-08-03 | 2018-04-04 | 마이오도파 리미티드 | L-dopa의 전신 합성 및 조절 |
EP3364997B1 (en) | 2015-10-22 | 2024-01-17 | University of Massachusetts | Aspartoacylase gene therapy in the treatment of canavan disease |
WO2017070516A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
EP3384035A4 (en) | 2015-12-02 | 2019-08-07 | Voyager Therapeutics, Inc. | ASSAYS FOR DETECTION OF NEUTRALIZING ANTIBODIES OF VAA |
WO2017136536A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
EP4094780A3 (en) | 2016-02-12 | 2023-02-08 | University of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
EP3440210A4 (en) | 2016-04-05 | 2019-11-27 | University of Massachusetts | COMPOSITIONS AND METHODS FOR SELECTIVE INHIBITION OF EXPRESSION OF GRAINHEAD PROTEIN |
WO2017181105A1 (en) | 2016-04-15 | 2017-10-19 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
KR20220108216A (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
IL262784B2 (en) | 2016-05-18 | 2023-10-01 | Voyager Therapeutics Inc | modulatory polynucleotides |
US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
CA3035522A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
EP3526333A4 (en) | 2016-10-13 | 2020-07-29 | University of Massachusetts | AAV CAPSIDE DESIGNS |
MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND TREATMENT METHODS FOR AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
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- 2010-11-09 RU RU2012123943A patent/RU2606012C2/ru not_active IP Right Cessation
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NZ600104A (en) | 2013-11-29 |
WO2011054976A2 (en) | 2011-05-12 |
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US20120309816A1 (en) | 2012-12-06 |
CA2779437A1 (en) | 2011-05-12 |
CN102695526A (zh) | 2012-09-26 |
EP2498825A2 (en) | 2012-09-19 |
RU2606012C2 (ru) | 2017-01-10 |
CL2012001215A1 (es) | 2012-08-03 |
WO2011054976A3 (en) | 2011-07-07 |
MX2012005450A (es) | 2012-08-15 |
EP2498825B1 (en) | 2017-03-29 |
RU2012123943A (ru) | 2013-12-20 |
JP5894535B2 (ja) | 2016-03-30 |
AU2010316982A1 (en) | 2012-06-14 |
JP2013509890A (ja) | 2013-03-21 |
US9593312B2 (en) | 2017-03-14 |
IL219704A0 (en) | 2012-07-31 |
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