AR127455A1 - Constructos de ácido nucleico, vectores víricos y partículas víricas - Google Patents
Constructos de ácido nucleico, vectores víricos y partículas víricasInfo
- Publication number
- AR127455A1 AR127455A1 ARP220102900A ARP220102900A AR127455A1 AR 127455 A1 AR127455 A1 AR 127455A1 AR P220102900 A ARP220102900 A AR P220102900A AR P220102900 A ARP220102900 A AR P220102900A AR 127455 A1 AR127455 A1 AR 127455A1
- Authority
- AR
- Argentina
- Prior art keywords
- nucleic acid
- viral
- acid construct
- stxbp1
- viral vector
- Prior art date
Links
- 102000039446 nucleic acids Human genes 0.000 title abstract 7
- 108020004707 nucleic acids Proteins 0.000 title abstract 7
- 150000007523 nucleic acids Chemical class 0.000 title abstract 7
- 239000013603 viral vector Substances 0.000 title abstract 5
- 239000002245 particle Substances 0.000 title abstract 4
- 230000003612 virological effect Effects 0.000 title abstract 4
- 102100025293 Syntaxin-binding protein 1 Human genes 0.000 abstract 6
- 101710096016 Syntaxin-binding protein 1 Proteins 0.000 abstract 6
- 108700019146 Transgenes Proteins 0.000 abstract 2
- 102000001708 Protein Isoforms Human genes 0.000 abstract 1
- 108010029485 Protein Isoforms Proteins 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000005714 functional activity Effects 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
Classifications
-
- 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
-
- 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
-
- 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
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
-
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
La presente invención se refiere a un constructo de ácido nucleico que comprende un transgén que codifica la proteína de unión a la sintaxina 1 (STXBP1, Munc-18), un vector vírico para encapsidado de dicho ácido nucleico en una partícula vírica; y el uso de dicha partícula vírica para tratar una enfermedad asociada con una pérdida de la actividad funcional de STXBP1. Reivindicación 1: Un constructo de ácido nucleico que comprende un transgén caracterizado porque codifica: i. una proteína de unión a la sintaxina 1 (STXBP1) que comprende la isoforma a, b, c, d, e, f, g o h, que tiene la secuencia dada en la SEQ ID Nº 9, 10, 11, 12, 13, 14, 15, o 16 respectivamente; o ii. una secuencia que tiene al menos 95% de identidad de secuencia con respecto a las SEQ ID Nº 9, 10, 11, 12, 13, 14, 15 o 16 y que mantiene la funcionalidad como STXBP1; o iii. una variante de origen natural que comprende, con referencia a la SEQ ID Nº 9, una o más mutaciones, como se observa en la Tabla 7. Reivindicación 6: Un vector vírico que comprende el constructo de ácido nucleico de acuerdo con una cualquiera de las reivindicaciones precedentes, caracterizado porque el vector vírico comprende, además, una repetición terminal invertida (ITR) en el flanco 5 y/o 3 de dicho constructo de ácido nucleico. Reivindicación 9: Una partícula vírica que comprende un constructo de ácido nucleico de acuerdo con una cualquiera de las reivindicaciones 1 a 5, o un vector vírico de acuerdo con una cualquiera de las reivindicaciones 6 a 8.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163263175P | 2021-10-28 | 2021-10-28 |
Publications (1)
Publication Number | Publication Date |
---|---|
AR127455A1 true AR127455A1 (es) | 2024-01-24 |
Family
ID=84362196
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP220102900A AR127455A1 (es) | 2021-10-28 | 2022-10-26 | Constructos de ácido nucleico, vectores víricos y partículas víricas |
Country Status (4)
Country | Link |
---|---|
AR (1) | AR127455A1 (es) |
CA (1) | CA3234666A1 (es) |
TW (1) | TW202325849A (es) |
WO (1) | WO2023073071A1 (es) |
Family Cites Families (18)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
WO1992001070A1 (en) | 1990-07-09 | 1992-01-23 | The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce | High efficiency packaging of mutant adeno-associated virus using amber suppressions |
US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
JP3534749B2 (ja) | 1991-08-20 | 2004-06-07 | アメリカ合衆国 | アデノウイルスが介在する胃腸管への遺伝子の輸送 |
EP1103610A1 (en) | 1999-11-26 | 2001-05-30 | Introgene B.V. | Production of vaccines from immortalised mammalian cell lines |
FR2808803B1 (fr) | 2000-05-11 | 2004-12-10 | Agronomique Inst Nat Rech | Cellules es modifiees et gene specifique de cellules es |
FR2836924B1 (fr) | 2002-03-08 | 2005-01-14 | Vivalis | Lignees de cellules aviaires utiles pour la production de substances d'interet |
EP1528101A1 (en) | 2003-11-03 | 2005-05-04 | ProBioGen AG | Immortalized avian cell lines for virus production |
FR2884255B1 (fr) | 2005-04-11 | 2010-11-05 | Vivalis | Utilisation de lignees de cellules souches aviaires ebx pour la production de vaccin contre la grippe |
EP1985305A1 (en) | 2007-04-24 | 2008-10-29 | Vivalis | Duck embryonic derived stem cell lines for the production of viral vaccines |
EP1995309A1 (en) | 2007-05-21 | 2008-11-26 | Vivalis | Recombinant protein production in avian EBx® cells |
WO2009084472A1 (ja) * | 2007-12-28 | 2009-07-09 | Public University Corporation Yokohama City University | 新生児期~乳児期発症の難治性てんかんの検出方法 |
WO2010020642A1 (en) * | 2008-08-20 | 2010-02-25 | Brainco Biopharma, S.L. | Stxbp1 as psychiatric biomarker in murine model system and their uses |
GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
TWI808079B (zh) * | 2017-04-03 | 2023-07-11 | 美商編碼製藥公司 | 組織選擇性轉基因表現 |
CN114929869A (zh) * | 2019-11-08 | 2022-08-19 | 科亚韦治疗公司 | 修饰的腺相关病毒载体及将其递送至中枢神经系统 |
EP4179098A1 (en) * | 2020-07-08 | 2023-05-17 | Baylor College Of Medicine | Gene therapy for stxbp1 encephalopathy |
WO2022214635A1 (en) * | 2021-04-08 | 2022-10-13 | Stichting Vu | Nucleic acid molecules for compensation of stxbp1 haploinsufficiency and their use in the treatment of stxbp1-related disorders |
-
2022
- 2022-10-26 WO PCT/EP2022/080020 patent/WO2023073071A1/en active Application Filing
- 2022-10-26 CA CA3234666A patent/CA3234666A1/en active Pending
- 2022-10-26 AR ARP220102900A patent/AR127455A1/es unknown
- 2022-10-28 TW TW111140997A patent/TW202325849A/zh unknown
Also Published As
Publication number | Publication date |
---|---|
WO2023073071A1 (en) | 2023-05-04 |
TW202325849A (zh) | 2023-07-01 |
CA3234666A1 (en) | 2023-05-04 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
AR108442A1 (es) | Proteínas de fusión gdf15 y usos de estas | |
PH12019500280A1 (en) | Trimer stabilizing hiv envelope protein mutations | |
CY1122800T1 (el) | Συνθεσεις του παραγοντα viii και μεθοδοι δημιουργιας και χρησης αυτων | |
ES2548030T3 (es) | Moléculas con semividas prolongadas y usos de las mismas | |
CO2018005306A2 (es) | Variantes del factor viii reducido con cpg, composiciones y métodos y usos para el tratamiento de trastornos de la hemostasia | |
EP4272765A3 (en) | Adeno-associated virus (aav) clade f vector and uses therefor | |
AR125394A2 (es) | Métodos y composiciones para la expresión génica en plantas | |
AU2018298422A1 (en) | Novel nucleic acid molecules | |
MX2019006467A (es) | Composiciones y métodos para mejorar la expresión génica. | |
ES2531290T3 (es) | Transferasas y oxidorreductasas, ácidos nucleicos que las codifican y métodos para prepararlas y usarlas | |
AR114339A1 (es) | Plataforma de presentación, productos y métodos con endosporas basadas en paenibacillus | |
AR057884A1 (es) | Plantas con caracteristicas de crecimiento mejoradas y un metodo para desarrollarlas | |
PE20200722A1 (es) | Metodos de terapia genica del factor viii (fviii) | |
MX2022005252A (es) | Composiciones de escherichia coli y metodos de las mismas. | |
CO2017011431A2 (es) | Proteínas de fusión de receptor de factor de crecimiento epidermal variante iii - mesotelina | |
PH12020500025A1 (en) | Trimer stabilizing hiv envelope protein mutations | |
BR112021017603A2 (pt) | Vírus adenoassociado (aav) mutante tendo propriedade de direcionamento para o cérebro | |
AR127455A1 (es) | Constructos de ácido nucleico, vectores víricos y partículas víricas | |
EA202190267A1 (ru) | Способы и композиции с использованием рекомбинантных дендритных клеток для терапии рака | |
AR061393A1 (es) | Componentes transportadores de nitrato | |
BR112022006842A2 (pt) | Construtos de igf2 variante | |
NO20085201L (no) | Rekombinante novirhabdoviruser og deres anvendelse | |
EA202090326A1 (ru) | Стабилизирующие тример мутации белка оболочки hiv | |
CO2023015564A2 (es) | Virus quimérico de la enfermedad de newcastle que expresa las proteínas hn y f del apmv | |
BR112023004874A2 (pt) | Construções de ácido nucléico, vetores virais e partículas virais |