HRP20200647T1 - Genski vektor - Google Patents
Genski vektor Download PDFInfo
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- HRP20200647T1 HRP20200647T1 HRP20200647TT HRP20200647T HRP20200647T1 HR P20200647 T1 HRP20200647 T1 HR P20200647T1 HR P20200647T T HRP20200647T T HR P20200647TT HR P20200647 T HRP20200647 T HR P20200647T HR P20200647 T1 HRP20200647 T1 HR P20200647T1
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- 239000013598 vector Substances 0.000 title claims 19
- 108090000623 proteins and genes Proteins 0.000 title claims 16
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 10
- 210000004027 cell Anatomy 0.000 claims 6
- 238000001415 gene therapy Methods 0.000 claims 6
- 108700019146 Transgenes Proteins 0.000 claims 5
- 108091070501 miRNA Proteins 0.000 claims 5
- 239000002679 microRNA Substances 0.000 claims 5
- 239000000825 pharmaceutical preparation Substances 0.000 claims 5
- 108091028066 Mir-126 Proteins 0.000 claims 3
- 102000006992 Interferon-alpha Human genes 0.000 claims 2
- 108010047761 Interferon-alpha Proteins 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 229940079593 drug Drugs 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 206010010099 Combined immunodeficiency Diseases 0.000 claims 1
- 102100025621 Cytochrome b-245 heavy chain Human genes 0.000 claims 1
- 108010042681 Galactosylceramidase Proteins 0.000 claims 1
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 claims 1
- 101001046686 Homo sapiens Integrin alpha-M Proteins 0.000 claims 1
- 102100022338 Integrin alpha-M Human genes 0.000 claims 1
- 208000028226 Krabbe disease Diseases 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 108010082739 NADPH Oxidase 2 Proteins 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- -1 c-Fes Proteins 0.000 claims 1
- 208000016532 chronic granulomatous disease Diseases 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 230000002132 lysosomal effect Effects 0.000 claims 1
- 230000004083 survival effect Effects 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/04—Immunostimulants
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/555—Interferons [IFN]
- C07K14/56—IFN-alpha
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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
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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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted 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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/027—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Epidemiology (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Claims (14)
1. Genski vektor za uporabu u genskoj terapiji koji sadrži najmanje jednu miRNA ciljanu sekvencu koja odgovara mir-130a ili mir-126 operativno povezanoj s transgenom koji kodira interferon-alfa, lizosomalni enzim galaktocerebrozidaze ili gp91 phox, naznačen time što se genski vektor koristi za sprječavanje ili smanjenje ekspresije transgena u krvotvornoj matičnoj stanici ili krvotvornoj progenitornoj stanici.
2. Genski vektor za uporabu prema patentnom zahtjevu 1 naznačen time što sadrži najmanje jednu miRNA ciljanu sekvencu koja odgovara mir-130a i najmanje jednu miRNA ciljanu sekvencu koja odgovara mir-126.
3. Genski vektor za uporabu prema patentnom zahtjevu 2, naznačen time što je broj kopija miRNA ciljane sekvence koja odgovara mir-130a dvostruki broj kopija miRNA ciljane sekvence koja odgovara mir-126.
4. Genski vektor za uporabu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što je vektor virusni vektor.
5. Genski vektor za uporabu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što se vektor može dobiti iz lentivirusa.
6. Genski vektor za uporabu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što vektor sadrži promotor specifičan za tkivo.
7. Genski vektor za uporabu prema patentnom zahtjevu 6, naznačen time što promotor specifičan za tkivo je CD11b, c-Fes, CYBB ili TEK promotor.
8. Genski vektor za uporabu prema bilo kojem prethodnom patentnom zahtjevu, naznačen time što transgen kodira interferon-alfa.
9. Farmaceutski pripravak za uporabu u genskoj terapiji koji sadrži genski vektor kako je definiran u bilo kojem od patentnih zahtjeva 1-8, naznačen time što se farmaceutski pripravak koristi za sprječavanje ili smanjenje ekspresije transgena krvotvorne matične stanice ili krvotvorne progenitorne stanice.
10. Izolirana stanica za uporabu u genskoj terapiji, naznačena time što je stanica zaražena ili transducirana s genskim vektorom kako je definiran u bilo kojem od patentnih zahtjeva 1-8, te pri čemu je stanica krvotvorna matična stanica ili progenitorna krvotvorna stanica.
11. Genski vektor kako je definiran u bilo kojem od patentnih zahtjeva 1-8, farmaceutski pripravak definiran u patentnom zahtjevu 9 ili stanica kako je definirana u patentnom zahtjevu 10 naznačeni time što su za uporabu za liječenje bolesti odabrane od leukodistrofije globoidnih stanica, kronične granulomatozne bolesti, ozbiljne kombinirane imunodeficijencije (SCID) i solidnih tumora.
12. Genski vektor kako je definiran u bilo kojem od patentnih zahtjeva 1-8, farmaceutski pripravak definiran u patentnom zahtjevu 9 ili stanica kako je definirana u patentnom zahtjevu 10 naznačeni time što su za use povećanje šansi za preživljavanje krvotvornih matičnih stanica ili krvotvornih progenitornih stanica vezanih za gensku terapiju.
13. Uporaba genskog vektora kako je definiran u bilo kojem od patentnih zahtjeva 1-8 ili farmaceutski pripravak definiran u patentnom zahtjevu 9 naznačena time što je za proizvodnju lijeka za gensku terapiju, pri čemu se genski vektor koristi za sprječavanje ili smanjenje ekspresije transgena u krvotvornoj matičnoj stanici ili krvotvornoj progenitornoj stanici.
14. Uporaba izolirane stanice kako je definirana u patentnom zahtjevu 10 naznačena time što je za proizvodnju lijeka za gensku terapiju.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US17412409P | 2009-04-30 | 2009-04-30 | |
| EP10723305.8A EP2424571B1 (en) | 2009-04-30 | 2010-04-30 | Gene vector |
| PCT/IB2010/001166 WO2010125471A2 (en) | 2009-04-30 | 2010-04-30 | Gene vector |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20200647T1 true HRP20200647T1 (hr) | 2020-10-02 |
Family
ID=42790671
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20200647TT HRP20200647T1 (hr) | 2009-04-30 | 2020-04-22 | Genski vektor |
Country Status (22)
| Country | Link |
|---|---|
| US (4) | US10287579B2 (hr) |
| EP (2) | EP3733214A1 (hr) |
| JP (2) | JP6144911B2 (hr) |
| KR (1) | KR101793615B1 (hr) |
| CN (1) | CN102596255B (hr) |
| AU (1) | AU2010243276B2 (hr) |
| BR (1) | BRPI1010873B1 (hr) |
| CA (1) | CA2759438C (hr) |
| CY (1) | CY1123218T1 (hr) |
| DK (1) | DK2424571T3 (hr) |
| EA (1) | EA023938B1 (hr) |
| ES (1) | ES2786039T3 (hr) |
| HR (1) | HRP20200647T1 (hr) |
| HU (1) | HUE049845T2 (hr) |
| IL (1) | IL215804A (hr) |
| LT (1) | LT2424571T (hr) |
| MX (1) | MX2011011508A (hr) |
| PL (1) | PL2424571T3 (hr) |
| PT (1) | PT2424571T (hr) |
| SG (2) | SG10201610827RA (hr) |
| SI (1) | SI2424571T1 (hr) |
| WO (1) | WO2010125471A2 (hr) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BRPI1010873B1 (pt) * | 2009-04-30 | 2020-04-14 | Fond Centro San Raffaele Del Monte Tabor | usos de um vetor de genes, de uma composição farmacêutica e de uma célula |
| ES2788198T3 (es) | 2010-05-14 | 2020-10-20 | Univ Oregon Health & Science | Vectores de CMVH y CMVRh recombinantes que codifican un antígeno heterólogo aislado del virus de la hepatitis B y usos de los mismos |
| EP2591095B1 (en) * | 2010-07-08 | 2016-04-27 | Duke University | Direct reprogramming of cells to cardiac myocyte fate |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| US20150190532A1 (en) * | 2012-04-04 | 2015-07-09 | The Trustees Of Columbia University In The City Of New York | Smooth muscle specific inhibition for anti-restenotic therapy |
| DK3401400T3 (da) | 2012-05-25 | 2019-06-03 | Univ California | Fremgangsmåder og sammensætninger til rna-styret mål-dna-modifikation og til rna-styret transskriptionsmodulering |
| JP6329537B2 (ja) | 2012-07-11 | 2018-05-23 | サンガモ セラピューティクス， インコーポレイテッド | 生物学的薬剤の送達のための方法および組成物 |
| JP6620018B2 (ja) | 2012-12-06 | 2019-12-11 | シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーＳｉｇｍａ−Ａｌｄｒｉｃｈ Ｃｏ．， ＬＬＣ | Ｃｒｉｓｐｒに基づくゲノム修飾および制御 |
| ES2959747T3 (es) | 2013-02-15 | 2024-02-28 | Bioverativ Therapeutics Inc | Gen del factor VIII optimizado |
| EP2971167B1 (en) | 2013-03-14 | 2019-07-31 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
| US10617721B2 (en) | 2013-10-24 | 2020-04-14 | Ospedale San Raffaele S.R.L. | Methods for genetic modification of stem cells |
| EP3160478A4 (en) | 2014-06-30 | 2018-05-16 | Bioverativ Therapeutics Inc. | Optimized factor ix gene |
| US10501811B2 (en) | 2015-04-22 | 2019-12-10 | Kyoto University | Method for sorting tissue cells |
| JP2018517421A (ja) * | 2015-06-15 | 2018-07-05 | マードック チルドレンズ リサーチ インスティチュート | キメリズムを測定する方法 |
| EP3359670B2 (en) | 2015-10-05 | 2024-02-14 | ModernaTX, Inc. | Methods for therapeutic administration of messenger ribonucleic acid drugs |
| US10188750B1 (en) | 2015-10-23 | 2019-01-29 | University Of South Florida | Self-replicating cell selective gene delivery compositions, methods, and uses thereof |
| LT3411478T (lt) | 2016-02-01 | 2022-09-26 | Bioverativ Therapeutics Inc. | Optimizuoti viii faktoriaus genai |
| GB201612214D0 (en) | 2016-07-14 | 2016-08-31 | Univ Oxford Innovation Ltd | Method |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| BR112019007982A2 (pt) * | 2016-10-18 | 2019-07-02 | Univ Oregon Health & Science | vetores de citomegalovírus elicitando células t restringidas por moléculas do complexo de histocompatibilidade e principal |
| CN106566838B (zh) * | 2016-11-14 | 2019-11-01 | 上海伯豪生物技术有限公司 | 一种基于CRISPR-Cas9技术的miR-126全长基因敲除试剂盒及其应用 |
| JP7252135B2 (ja) * | 2017-04-21 | 2023-04-04 | オスペダーレ サン ラファエレ エス．アール．エル | 遺伝子療法 |
| IT201700086403A1 (it) * | 2017-07-27 | 2019-01-27 | Univ Del Piemonte Orientale | Endothelial-specific promoter sequences and uses thereof |
| TW201920255A (zh) | 2017-08-09 | 2019-06-01 | 美商生物化學醫療公司 | 核酸分子及其用途 |
| WO2019152692A1 (en) | 2018-02-01 | 2019-08-08 | Bioverativ Therapeutics, Inc. | Use of lentiviral vectors expressing factor viii |
| BR112021002017A2 (pt) | 2018-08-09 | 2021-05-11 | Bioverativ Therapeutics Inc. | moléculas de ácido nucleico e usos das mesmas para terapia genética não viral |
| CN113396223A (zh) | 2018-12-06 | 2021-09-14 | 比奥维拉迪维治疗股份有限公司 | 表达因子ix的慢病毒载体的用途 |
| WO2021067389A1 (en) | 2019-09-30 | 2021-04-08 | Bioverativ Therapeutics Inc. | Lentiviral vector formulations |
| US20220378937A1 (en) * | 2019-11-12 | 2022-12-01 | The Regents Of The University Of California | Lentiviral vectors in hematopoietic stem cells to treat x-linked chronic granulomatous disease |
| KR20230074703A (ko) | 2020-06-24 | 2023-05-31 | 바이오버라티브 테라퓨틱스 인크. | 유리 인자 viii을 상기 단백질이 발현되도록 변형된 렌티바이러스 벡터의 제제로부터 제거하는 방법 |
| CA3189673A1 (en) | 2020-08-23 | 2022-03-03 | Ajay MAGHODIA | Modified baculovirus system for improved production of closed-ended dna (cedna) |
| TW202323274A (zh) | 2021-08-23 | 2023-06-16 | 美商百歐維拉提夫治療公司 | 優化因子viii基因 |
| EP4392566A2 (en) | 2021-08-23 | 2024-07-03 | Bioverativ Therapeutics Inc. | Baculovirus expression system |
| CN118019758A (zh) | 2021-09-30 | 2024-05-10 | 比奥维拉迪维治疗股份有限公司 | 编码免疫原性降低的因子viii多肽的核酸 |
| WO2024026257A2 (en) * | 2022-07-25 | 2024-02-01 | Modernatx, Inc. | Engineered polynucleotides for cell selective expression |
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| KR20130114758A (ko) * | 2005-05-27 | 2013-10-17 | 오스페달레 산 라파엘 에스.알.엘. | Ｍｉ-ｒｎａ를 포함하는 유전자 벡터 |
| WO2006128245A1 (en) * | 2005-06-03 | 2006-12-07 | Southern Adelaide Health Service-Flinders Medical Centre | Targeting cells with altered microrna expression |
| US20090180989A1 (en) * | 2005-10-05 | 2009-07-16 | Synageva Biopharma Corp. | Compositions and methods for delivering nucleotide sequences to vertebrates |
| EP2468890B1 (en) | 2006-01-05 | 2014-03-19 | The Ohio State University Research Foundation | MicroRNA-based methods and compositions for the diagnosis and treatment of solid cancers of the breast or lung |
| US20080095750A1 (en) | 2006-05-10 | 2008-04-24 | Pennington Biomedical Research Center, Louisiana State University System | Use of adipose-derived stem cells for treatment of leukodystrophies |
| EP2104736B1 (en) | 2006-12-08 | 2011-11-23 | Asuragen, INC. | Mir-126 regulated genes and pathways as targets for therapeutic intervention |
| LT2158322T (lt) | 2007-06-06 | 2017-08-25 | Genzyme Corporation | Lizosomų kaupimo ligų genų terapija |
| WO2010055413A1 (en) | 2008-11-12 | 2010-05-20 | Fondazione Centro San Raffaele Del Monte Tabor | Gene vector for inducing transgene-specific immune tolerance |
| BRPI1010873B1 (pt) | 2009-04-30 | 2020-04-14 | Fond Centro San Raffaele Del Monte Tabor | usos de um vetor de genes, de uma composição farmacêutica e de uma célula |
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