MX377804B - Composiciones utiles en el tratamiento de deficiencia de la ornitina transcarbamilasa (otc). - Google Patents
Composiciones utiles en el tratamiento de deficiencia de la ornitina transcarbamilasa (otc).Info
- Publication number
- MX377804B MX377804B MX2016011686A MX2016011686A MX377804B MX 377804 B MX377804 B MX 377804B MX 2016011686 A MX2016011686 A MX 2016011686A MX 2016011686 A MX2016011686 A MX 2016011686A MX 377804 B MX377804 B MX 377804B
- Authority
- MX
- Mexico
- Prior art keywords
- treatment
- deficiency
- ornithine transcarbamylase
- otc
- compositions useful
- Prior art date
Links
- 102000007981 Ornithine carbamoyltransferase Human genes 0.000 title 2
- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 title 2
- 230000007812 deficiency Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 101000986595 Homo sapiens Ornithine transcarbamylase, mitochondrial Proteins 0.000 abstract 2
- 102100028200 Ornithine transcarbamylase, mitochondrial Human genes 0.000 abstract 2
- 208000019425 cirrhosis of liver Diseases 0.000 abstract 2
- 201000011278 ornithine carbamoyltransferase deficiency Diseases 0.000 abstract 2
- 206010020575 Hyperammonaemia Diseases 0.000 abstract 1
- 208000000599 Ornithine Carbamoyltransferase Deficiency Disease Diseases 0.000 abstract 1
- 206010052450 Ornithine transcarbamoylase deficiency Diseases 0.000 abstract 1
- 208000035903 Ornithine transcarbamylase deficiency Diseases 0.000 abstract 1
- 230000004761 fibrosis Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 208000024891 symptom Diseases 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
Classifications
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- 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
- 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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- 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
- C12N9/10—Transferases (2.)
- C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
- C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y201/00—Transferases transferring one-carbon groups (2.1)
- C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
- C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)
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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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10343—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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—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
- 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
- C12N2750/14032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
- 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
- C12N2750/14033—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
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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
- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2750/14071—Demonstrated in vivo effect
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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
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461950157P | 2014-03-09 | 2014-03-09 | |
| PCT/US2015/019513 WO2015138348A1 (en) | 2014-03-09 | 2015-03-09 | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MX2016011686A MX2016011686A (es) | 2016-11-07 |
| MX377804B true MX377804B (es) | 2025-03-11 |
Family
ID=52737408
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2016011686A MX377804B (es) | 2014-03-09 | 2015-03-09 | Composiciones utiles en el tratamiento de deficiencia de la ornitina transcarbamilasa (otc). |
Country Status (22)
| Country | Link |
|---|---|
| US (6) | US10167454B2 (https=) |
| EP (2) | EP3116900B1 (https=) |
| JP (2) | JP6822841B2 (https=) |
| KR (1) | KR102390075B1 (https=) |
| AU (2) | AU2015229750A1 (https=) |
| BR (2) | BR122023023004A2 (https=) |
| CA (1) | CA2939950C (https=) |
| CL (2) | CL2016002235A1 (https=) |
| CY (1) | CY1123378T1 (https=) |
| DK (1) | DK3116900T3 (https=) |
| ES (1) | ES2821938T3 (https=) |
| HR (1) | HRP20201544T1 (https=) |
| HU (1) | HUE051311T2 (https=) |
| IL (2) | IL247329B (https=) |
| LT (1) | LT3116900T (https=) |
| MX (1) | MX377804B (https=) |
| PL (1) | PL3116900T3 (https=) |
| PT (1) | PT3116900T (https=) |
| RS (1) | RS60902B1 (https=) |
| SI (1) | SI3116900T1 (https=) |
| SM (1) | SMT202000531T1 (https=) |
| WO (2) | WO2015138348A1 (https=) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX374963B (es) | 2011-04-29 | 2025-03-06 | Selecta Biosciences Inc | Nanoportadores sintéticos tolerogénicos para reducir las respuestas de anticuerpos. |
| BR122020023215B1 (pt) | 2013-05-03 | 2022-11-22 | Selecta Biosciences, Inc | Composição e kit de nanocarreadores sintéticos tolerogênicos para reduzir ou prevenir anafilaxia em resposta a um antígeno não alergênico |
| AU2014296278C1 (en) | 2013-07-30 | 2023-02-02 | Genevant Sciences Gmbh | Block copolymers and their conjugates or complexes with oligonucleotides |
| SMT202000531T1 (it) | 2014-03-09 | 2020-11-10 | Univ Pennsylvania | Composizioni utili in trattamento di deficit di ornitina transcarbamilasi(otc) |
| JP7242180B2 (ja) | 2014-09-07 | 2023-03-20 | セレクタ バイオサイエンシーズ インコーポレーテッド | 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物 |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| JP2018509387A (ja) | 2015-01-21 | 2018-04-05 | フェーズアールエックス インコーポレイテッド | 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム |
| WO2017059902A1 (en) | 2015-10-07 | 2017-04-13 | Biontech Rna Pharmaceuticals Gmbh | 3' utr sequences for stabilization of rna |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| IL315358A (en) | 2016-08-18 | 2024-11-01 | Univ California | CRISPR-CAS genome engineering using a modular AAV delivery system |
| MA46761A (fr) * | 2016-11-10 | 2019-09-18 | Translate Bio Inc | Administration sous-cutanée d'arn messager |
| EP3562510A4 (en) | 2016-12-30 | 2021-01-06 | Genevant Sciences GmbH | BRANCHED PEG MOLECULES AND ASSOCIATED COMPOSITIONS AND PROCEDURES |
| KR102738752B1 (ko) * | 2016-12-30 | 2024-12-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
| WO2018127382A1 (en) * | 2017-01-03 | 2018-07-12 | Ethris Gmbh | Ornithine transcarbamylase coding polyribonucleotides and formulations thereof |
| PT3568483T (pt) * | 2017-01-10 | 2025-11-18 | Childrens Medical Res Institute | Polinucleótidos e vetores para a expressão de transgenes |
| ES3036482T3 (en) | 2017-03-11 | 2025-09-19 | Cartesian Therapeutics Inc | Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant |
| JP7119009B2 (ja) | 2017-05-31 | 2022-08-16 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 最適化されたヒト凝固第ｉｘ因子遺伝子発現カセットおよびそれらの使用 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| NZ762100A (en) | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
| CA3079428A1 (en) * | 2017-11-22 | 2019-05-31 | Modernatx, Inc. | Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders |
| TW202016314A (zh) | 2018-05-09 | 2020-05-01 | 美商拜奧馬林製藥公司 | 治療苯丙酮尿症之方法 |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| AU2019271132B2 (en) * | 2018-05-15 | 2026-02-05 | Translate Bio, Inc. | Subcutaneous delivery of messenger RNA |
| KR20210019996A (ko) | 2018-05-15 | 2021-02-23 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 조성물 및 방법 |
| WO2020018583A1 (en) | 2018-07-16 | 2020-01-23 | Selecta Biosciences, Inc. | Methods and compositions of otc constructs and vectors |
| EP3823981A4 (en) | 2018-07-17 | 2022-04-06 | Neuromyon Inc. | TREATMENT OF NEUROPATHY WITH DNA CONSTRUCTS EXPRESSING IGF-1 ISOFORMS |
| EP3833766A1 (en) * | 2018-08-09 | 2021-06-16 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| AU2019394996B2 (en) * | 2018-12-06 | 2025-11-06 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| WO2020160508A1 (en) | 2019-01-31 | 2020-08-06 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of aav capsids |
| AU2020287627A1 (en) | 2019-06-04 | 2021-11-18 | Selecta Biosciences, Inc. | Formulations and doses of PEGylated uricase |
| US12351834B2 (en) | 2020-03-03 | 2025-07-08 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| AU2021230361A1 (en) * | 2020-03-04 | 2022-09-08 | Poseida Therapeutics, Inc. | Compositions and methods for the treatment of Metabolic Liver Disorders |
| US20230235353A1 (en) * | 2020-03-19 | 2023-07-27 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| MX2022014258A (es) | 2020-05-12 | 2023-02-22 | Univ Pennsylvania | Composiciones para la reducción específica de un fármaco de la expresión de transgén. |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2022224372A1 (ja) * | 2021-04-21 | 2022-10-27 | 学校法人自治医科大学 | オルニチントランスカルバミラーゼ欠損症の治療用アデノ随伴ウイルスビリオン |
| EP4392445A1 (en) * | 2021-08-23 | 2024-07-03 | Bioverativ Therapeutics Inc. | Closed-end dna production with inverted terminal repeat sequences |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2023140971A1 (en) * | 2022-01-21 | 2023-07-27 | The Trustees Of The University Ofpennsylvania | Methods for treatment of ornithine transcarbamylase (otc) deficiency |
| EP4602157A2 (en) * | 2022-10-11 | 2025-08-20 | Pacific Northwest Research Institute | Constructs relating to genetic disorders |
| WO2025229995A1 (ja) * | 2024-04-30 | 2025-11-06 | 学校法人自治医科大学 | 核酸導入用ベクター、核酸導入方法、ポリヌクレオチド、及びベクター |
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| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| US5652224A (en) | 1995-02-24 | 1997-07-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism |
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