AR114438A1 - VECTORS FOR GENETIC THERAPIES AND METHODS FOR THEIR USE - Google Patents
VECTORS FOR GENETIC THERAPIES AND METHODS FOR THEIR USEInfo
- Publication number
- AR114438A1 AR114438A1 ARP190101137A ARP190101137A AR114438A1 AR 114438 A1 AR114438 A1 AR 114438A1 AR P190101137 A ARP190101137 A AR P190101137A AR P190101137 A ARP190101137 A AR P190101137A AR 114438 A1 AR114438 A1 AR 114438A1
- Authority
- AR
- Argentina
- Prior art keywords
- gene therapy
- nucleic acid
- vector
- methods
- peptide
- Prior art date
Links
Classifications
-
- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
- Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
- Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
Landscapes
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Vectores para terapias genéticas y métodos para su uso mejorados que, en algunas formas de realización, comprenden secuencias para una expresión y direccionamiento celulares mejorados de una proteína terapéutica. Reivindicación 1: Un vector para terapia génica caracterizado porque comprende una construcción de ácido nucleico que comprende: (a) una secuencia de ácidos nucleicos que codifica para una proteína terapéutica, y (b) una secuencia de ácidos nucleicos que codifica para un péptido que se une al receptor de manosa 6-fosfato independiente de cationes (MP6) (CI-MPR) con alta afinidad. Reivindicación 2: El vector para terapia génica de la reivindicación 1, caracterizado porque el péptido es una variante de péptido IGF2 (vlGF2). Reivindicación 12: El vector para terapia génica de la reivindicación 11, caracterizado porque el trastorno genético es un trastorno de almacenamiento lisosomal.Improved gene therapy vectors and methods for their use which, in some embodiments, comprise sequences for improved cellular expression and targeting of a therapeutic protein. Claim 1: A vector for gene therapy characterized in that it comprises a nucleic acid construct that comprises: (a) a nucleic acid sequence that codes for a therapeutic protein, and (b) a nucleic acid sequence that codes for a peptide that is binds to the cation-independent mannose 6-phosphate receptor (MP6) (CI-MPR) with high affinity. Claim 2: The gene therapy vector of claim 1, characterized in that the peptide is an IGF2 peptide variant (vlGF2). Claim 12: The vector for gene therapy of claim 11, characterized in that the genetic disorder is a lysosomal storage disorder.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862664741P | 2018-04-30 | 2018-04-30 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR114438A1 true AR114438A1 (en) | 2020-09-02 |
Family
ID=72670891
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP190101137A AR114438A1 (en) | 2018-04-30 | 2019-04-30 | VECTORS FOR GENETIC THERAPIES AND METHODS FOR THEIR USE |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR114438A1 (en) |
-
2019
- 2019-04-30 AR ARP190101137A patent/AR114438A1/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| EA201990861A1 (en) | RNA-GUIDED MODIFICATING NUCLEIC ACIDS ENZYMES AND METHODS OF APPLICATION | |
| AR080229A1 (en) | FIBRONECTINE ARMAZON DOMAIN PROTEINS THAT JOIN IL -23 (INTERLEUCINE 23) | |
| AR109680A1 (en) | RECOMBINANT PROTEINS AND THEIR USES | |
| MX2022006188A (en) | Adeno-associated viral vector variants. | |
| AR108442A1 (en) | GDF15 FUSION PROTEINS AND USES OF THESE | |
| GT201700162A (en) | ANTI-CD3 ANTIBODIES, ANTI-CD123 ANTIBODIES AND BIESPECIFIC ANTIBODIES THAT SPECIFICALLY JOIN CD3 AND / OR CD123 | |
| CO6180445A2 (en) | ANTI NOTCH3 AGONIST ANTIBODIES AND THEIR USE IN THE TREATMENT OF DISEASES RELATED TO THE NOTCH3 GENE | |
| DOP2019000011A (en) | BISPECIFIC ANTIBODY TYPE UNION PROTEINS THAT SPECIFICALLY JOIN CD3 AND CD123 | |
| MX2022015081A (en) | Hsd17b13 variants and uses thereof. | |
| MX2020011514A (en) | Gene therapy constructs and methods of use. | |
| AR047729A1 (en) | THERAPEUTIC ANTIBODIES THAT BIND APOLIPOPROTEIN E (APOE) | |
| MX2021004391A (en) | Intein proteins and uses thereof. | |
| CO2020010269A2 (en) | Fc variants with improved fcrn binding and long half-life | |
| PE20171336A1 (en) | ANTIBODIES AGAINST TAU AND ITS USES | |
| CL2019003404A1 (en) | Isolated tumor associated peptide (tumap); nucleic acid; expression vector; recombinant host cell comprising the peptide; method of producing the peptide; use to prepare a rare useful drug to treat cancer; pharmaceutical kit. (divisional application 201702407) | |
| MX2022005916A (en) | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders. | |
| BR112022008214A2 (en) | COMPOSITIONS AND METHODS TO TREAT NEUROSENSORY HEARING LOSS USING OTOFERLIN DUAL VECTOR SYSTEMS | |
| BR112022006842A2 (en) | VARIANT IGF2 CONSTRUCTS | |
| EA201691011A1 (en) | SEQUENCE OF MODIFIED ENDOLYSINE EL188 | |
| EA201691981A1 (en) | IMPROVED TRANSPORT MOLECULES OF THE MODULAR ANTIGEN AND THEIR APPLICATION | |
| EA201691004A1 (en) | SEQUENCE OF MODIFIED ENDOLYSIN KZ144 | |
| EA202190267A1 (en) | METHODS AND COMPOSITIONS USING RECOMBINANT DENDRITIC CELLS FOR CANCER THERAPY | |
| PE20170941A1 (en) | HERBICIDE TOLERANCE GENES AND METHODS TO USE THEM | |
| AR114438A1 (en) | VECTORS FOR GENETIC THERAPIES AND METHODS FOR THEIR USE | |
| CO2019012204A2 (en) | Anti-jagged1 antigen-binding proteins |