TN2016000460A1 - Aav vectors for retinal and cns gene therapy. - Google Patents
Aav vectors for retinal and cns gene therapy.Info
- Publication number
- TN2016000460A1 TN2016000460A1 TN2016000460A TN2016000460A TN2016000460A1 TN 2016000460 A1 TN2016000460 A1 TN 2016000460A1 TN 2016000460 A TN2016000460 A TN 2016000460A TN 2016000460 A TN2016000460 A TN 2016000460A TN 2016000460 A1 TN2016000460 A1 TN 2016000460A1
- Authority
- TN
- Tunisia
- Prior art keywords
- cns
- raav
- gene therapy
- improved
- disorders
- 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
- 238000000034 method Methods 0.000 abstract 4
- 208000022873 Ocular disease Diseases 0.000 abstract 2
- 208000015114 central nervous system disease Diseases 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 208000035475 disorder Diseases 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 239000002245 particle Substances 0.000 abstract 2
- 239000013646 rAAV2 vector Substances 0.000 abstract 2
- 108091016585 CD44 antigen Proteins 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 102000008055 Heparan Sulfate Proteoglycans Human genes 0.000 abstract 1
- 229920002971 Heparan sulfate Polymers 0.000 abstract 1
- 108090000054 Syndecan-2 Proteins 0.000 abstract 1
- 150000001413 amino acids Chemical class 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
Provided herein are improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of ocular disorders or CNS disorders wherein the rAAV comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan. The invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases with improved compositions of rAAV particles. Further provided herein are improved recombinant adeno-associated virus (rAAV) (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of disorders of the CNS. The invention provides methods for delivering the rAAV to the CNS, methods for treating disorders of the CNS with improved compositions of rAAV particles, and kits for delivering the rAAV to the CNS and/or treating a CNS 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 (1)
| Publication Number | Publication Date |
|---|---|
| TN2016000460A1 true TN2016000460A1 (en) | 2018-04-04 |
Family
ID=58700040
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TN2016000460A TN2016000460A1 (en) | 2014-05-02 | 2015-05-02 | Aav vectors for retinal and cns 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 unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AR100274A1 (en) | 2016-09-21 |
| MA39439B2 (en) | 2020-11-30 |
| MA39439A1 (en) | 2018-06-29 |
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