AR115858A1 - TREATMENT OF MUCOPOLYSACARIDOSIS IVA - Google Patents
TREATMENT OF MUCOPOLYSACARIDOSIS IVAInfo
- Publication number
- AR115858A1 AR115858A1 ARP190102119A ARP190102119A AR115858A1 AR 115858 A1 AR115858 A1 AR 115858A1 AR P190102119 A ARP190102119 A AR P190102119A AR P190102119 A ARP190102119 A AR P190102119A AR 115858 A1 AR115858 A1 AR 115858A1
- Authority
- AR
- Argentina
- Prior art keywords
- iva
- treatment
- mucopolysacaridosis
- raavs
- gene therapy
- Prior art date
Links
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 abstract 3
- 238000000034 method Methods 0.000 abstract 3
- 101001066305 Homo sapiens N-acetylgalactosamine-6-sulfatase Proteins 0.000 abstract 2
- 238000001415 gene therapy Methods 0.000 abstract 2
- 208000012253 mucopolysaccharidosis IVA Diseases 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 208000025797 Mucopolysaccharidosis type 4A Diseases 0.000 abstract 1
- 102100031688 N-acetylgalactosamine-6-sulfatase Human genes 0.000 abstract 1
- 210000000988 bone and bone Anatomy 0.000 abstract 1
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 abstract 1
Landscapes
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
Abstract
Métodos de genoterapia para el tratamiento de la mucopolisacaridosis tipo IVA (MPS IVA), que incluyen el uso de virus adenoasocidados recombinantes (rAAV) para administrar N-acetilgalactosamina-6-sulfato sulfatasa humana (hGALNS) al hueso de un sujeto humano al que le han diagnosticado MPS IVA. También rAAV que se pueden usar en los métodos de genoterapia, y métodos para elaborar tales rAAV.Gene therapy methods for the treatment of mucopolysaccharidosis type IVA (MPS IVA), which include the use of recombinant adeno-associated virus (rAAV) to deliver human N-acetylgalactosamine-6-sulfate sulfatase (hGALNS) to the bone of a human subject to whom have diagnosed MPS IVA. Also rAAVs that can be used in gene therapy methods, and methods for making such rAAVs.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862711238P | 2018-07-27 | 2018-07-27 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR115858A1 true AR115858A1 (en) | 2021-03-03 |
Family
ID=75905094
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP190102119A AR115858A1 (en) | 2018-07-27 | 2019-07-26 | TREATMENT OF MUCOPOLYSACARIDOSIS IVA |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR115858A1 (en) |
-
2019
- 2019-07-26 AR ARP190102119A patent/AR115858A1/en not_active Application Discontinuation
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA | Abandonment or withdrawal |