MX2021002418A - Genes sinteticos activados por retroalimentacion, casetes de coincidencia de semilla objetivo y sus usos. - Google Patents
Genes sinteticos activados por retroalimentacion, casetes de coincidencia de semilla objetivo y sus usos.Info
- Publication number
- MX2021002418A MX2021002418A MX2021002418A MX2021002418A MX2021002418A MX 2021002418 A MX2021002418 A MX 2021002418A MX 2021002418 A MX2021002418 A MX 2021002418A MX 2021002418 A MX2021002418 A MX 2021002418A MX 2021002418 A MX2021002418 A MX 2021002418A
- Authority
- MX
- Mexico
- Prior art keywords
- cassettes
- synthetic genes
- target seed
- seed match
- enabled synthetic
- Prior art date
Links
- 108700005078 Synthetic Genes Proteins 0.000 title abstract 2
- 108700019146 Transgenes Proteins 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
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
- A61K48/005—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
-
- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
-
- 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
-
- 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
- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
- C07K14/4703—Inhibitors; Suppressors
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
-
- 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
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (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)
Abstract
Esta invención se refiere a genes sintéticos habilitados por retroalimentación, casetes de polinucleótidos objetivo, vectores y composiciones farmacéuticas para el propósito de proveer expresión de transgen en tejidos objetivo que es capaz de regulación endógena para tratar trastornos tales como trastornos de capacidad intelectual sensible a dosis, así como métodos de elaboración y métodos para utilizarlos.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862725126P | 2018-08-30 | 2018-08-30 | |
US201962861044P | 2019-06-13 | 2019-06-13 | |
PCT/US2019/048776 WO2020047234A1 (en) | 2018-08-30 | 2019-08-29 | Feedback enabled synthetic genes, target seed match cassettes, and their uses |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2021002418A true MX2021002418A (es) | 2021-04-28 |
Family
ID=69643735
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2021002418A MX2021002418A (es) | 2018-08-30 | 2019-08-29 | Genes sinteticos activados por retroalimentacion, casetes de coincidencia de semilla objetivo y sus usos. |
Country Status (12)
Country | Link |
---|---|
US (1) | US20210170051A1 (es) |
EP (1) | EP3844277A4 (es) |
JP (1) | JP2021534778A (es) |
KR (1) | KR20210039495A (es) |
CN (1) | CN113056560B (es) |
AU (1) | AU2019333140A1 (es) |
BR (1) | BR112021003469A2 (es) |
CA (1) | CA3110289A1 (es) |
IL (1) | IL280956A (es) |
MX (1) | MX2021002418A (es) |
SG (1) | SG11202102013XA (es) |
WO (1) | WO2020047234A1 (es) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP4055174A4 (en) * | 2019-11-08 | 2024-01-03 | Univ Texas | TRANSGENIC CASSETTES, AAV VECTORS AND AAV VIRAL VECTORS FOR EXPRESSION OF THE SLC6A1 GENE WITH OPTIMIZED HUMAN CODONS |
IL293431A (en) * | 2019-12-05 | 2022-07-01 | Univ Texas | Transgene cassettes designed to express human mecp2 gene |
Family Cites Families (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20110172295A1 (en) * | 2008-04-11 | 2011-07-14 | The University Of North Carolina At Chapel Hill | METHODS AND COMPOSITIONS FOR THE REGULATION OF microRNA PROCESSING |
US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
US11219696B2 (en) * | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
EA201492116A1 (ru) * | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
CN103045596A (zh) * | 2012-12-20 | 2013-04-17 | 徐勇 | 唐氏综合征21号染色体相关miRNA、基因、筛选方法和应用 |
JP2016504050A (ja) * | 2013-01-17 | 2016-02-12 | モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. | 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド |
WO2016145014A1 (en) * | 2015-03-10 | 2016-09-15 | President And Fellows Of Harvard College | Methods for treatment of autism spectrum disorders |
US20210101938A1 (en) * | 2017-03-24 | 2021-04-08 | The University Court Of The University Of Edinburgh | Mecp2 based therapy |
IL293431A (en) * | 2019-12-05 | 2022-07-01 | Univ Texas | Transgene cassettes designed to express human mecp2 gene |
-
2019
- 2019-08-29 CN CN201980070905.7A patent/CN113056560B/zh active Active
- 2019-08-29 KR KR1020217008991A patent/KR20210039495A/ko active Search and Examination
- 2019-08-29 WO PCT/US2019/048776 patent/WO2020047234A1/en unknown
- 2019-08-29 EP EP19853521.3A patent/EP3844277A4/en active Pending
- 2019-08-29 BR BR112021003469-2A patent/BR112021003469A2/pt not_active Application Discontinuation
- 2019-08-29 JP JP2021510432A patent/JP2021534778A/ja active Pending
- 2019-08-29 CA CA3110289A patent/CA3110289A1/en not_active Abandoned
- 2019-08-29 MX MX2021002418A patent/MX2021002418A/es unknown
- 2019-08-29 US US17/271,432 patent/US20210170051A1/en active Pending
- 2019-08-29 SG SG11202102013XA patent/SG11202102013XA/en unknown
- 2019-08-29 AU AU2019333140A patent/AU2019333140A1/en active Pending
-
2021
- 2021-02-18 IL IL280956A patent/IL280956A/en unknown
Also Published As
Publication number | Publication date |
---|---|
EP3844277A4 (en) | 2022-06-22 |
KR20210039495A (ko) | 2021-04-09 |
US20210170051A1 (en) | 2021-06-10 |
AU2019333140A1 (en) | 2021-03-18 |
IL280956A (en) | 2021-04-29 |
BR112021003469A2 (pt) | 2021-05-18 |
CN113056560A (zh) | 2021-06-29 |
WO2020047234A9 (en) | 2020-05-14 |
EP3844277A1 (en) | 2021-07-07 |
SG11202102013XA (en) | 2021-03-30 |
CA3110289A1 (en) | 2020-03-05 |
CN113056560B (zh) | 2024-05-07 |
JP2021534778A (ja) | 2021-12-16 |
WO2020047234A1 (en) | 2020-03-05 |
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