MA39439B2 - Aav vectors for retinal and snc gene therapy - Google Patents
Aav vectors for retinal and snc gene therapyInfo
- Publication number
- MA39439B2 MA39439B2 MA39439A MA39439A MA39439B2 MA 39439 B2 MA39439 B2 MA 39439B2 MA 39439 A MA39439 A MA 39439A MA 39439 A MA39439 A MA 39439A MA 39439 B2 MA39439 B2 MA 39439B2
- Authority
- MA
- Morocco
- Prior art keywords
- snc
- raav
- improved
- gene therapy
- relates
- Prior art date
Links
- 238000001415 gene therapy Methods 0.000 title abstract 3
- 230000002207 retinal effect Effects 0.000 title abstract 2
- 239000013598 vector Substances 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 4
- 238000000034 method Methods 0.000 abstract 3
- 208000030533 eye disease Diseases 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 239000002245 particle Substances 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 229920002971 Heparan sulfate Polymers 0.000 abstract 1
- QAOWNCQODCNURD-UHFFFAOYSA-L Sulfate Chemical compound [O-]S([O-])(=O)=O QAOWNCQODCNURD-UHFFFAOYSA-L 0.000 abstract 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 238000006467 substitution reaction Methods 0.000 abstract 1
- 230000026683 transduction Effects 0.000 abstract 1
- 238000010361 transduction Methods 0.000 abstract 1
Landscapes
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
La présente invention concerne des raav améliorés (par exemple, raav2 raavrh8r, etc) pour une thérapie génique améliorée de troubles oculaires ou de troubles du snc, lesquels raav comprennent une ou plusieurs substitutions d'acides aminés qui interagissent avec un protéoglycane à sulfate d'héparane. L'invention concerne la transduction améliorée de cellules rétiniennes et des procédés de traitement de maladies oculaires avec des compositions améliorées de particules de raav. En outre l'invention concerne des virus adéno-associés recombinés (raav) (par exemple, raav2 raavrh8r, etc) pour une thérapie génique améliorée de troubles du snc. L'invention concerne des procédés d'administration de raav au snc, des procédés de traitement de troubles du snc avec les compositions améliorées de particules de raav, et des kits pour administrer le raav au snc et/ou traiter un trouble du snc.The present invention relates to improved raavs (e.g. raav2 raavrh8r, etc.) for improved gene therapy of eye disorders or snc disorders, which raav comprises one or more amino acid substitutions which interact with a sulfate proteoglycan. heparan. The invention relates to improved transduction of retinal cells and methods of treating eye diseases with improved compositions of raav particles. Further, the invention relates to recombinant adeno-associated viruses (raav) (eg, raav2 raavrh8r, etc.) for improved gene therapy of snc disorders. The invention relates to methods of delivering raav to snc, methods of treating snc disorders with the improved raav particle compositions, and kits for administering raav to snc and / or treating an snc disorder.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461988131P | 2014-05-02 | 2014-05-02 | |
| PCT/US2015/028966 WO2015168666A2 (en) | 2014-05-02 | 2015-05-02 | Aav vectors for retinal and cns gene therapy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MA39439A1 MA39439A1 (en) | 2018-06-29 |
| MA39439B2 true MA39439B2 (en) | 2020-11-30 |
Family
ID=58700040
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MA39439A MA39439B2 (en) | 2014-05-02 | 2015-05-02 | Aav vectors for retinal and snc gene therapy |
Country Status (3)
| Country | Link |
|---|---|
| AR (1) | AR100274A1 (en) |
| MA (1) | MA39439B2 (en) |
| TN (1) | TN2016000460A1 (en) |
-
2015
- 2015-05-02 MA MA39439A patent/MA39439B2/en unknown
- 2015-05-02 TN TN2016000460A patent/TN2016000460A1/en unknown
- 2015-05-04 AR ARP150101342A patent/AR100274A1/en active IP Right Grant
Also Published As
| Publication number | Publication date |
|---|---|
| AR100274A1 (en) | 2016-09-21 |
| MA39439A1 (en) | 2018-06-29 |
| TN2016000460A1 (en) | 2018-04-04 |
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