AR118584A1 - RECOMBINANT ADENO-ASSOCIATED VIRUSES AND THEIR USES - Google Patents
RECOMBINANT ADENO-ASSOCIATED VIRUSES AND THEIR USESInfo
- Publication number
- AR118584A1 AR118584A1 ARP200100940A ARP200100940A AR118584A1 AR 118584 A1 AR118584 A1 AR 118584A1 AR P200100940 A ARP200100940 A AR P200100940A AR P200100940 A ARP200100940 A AR P200100940A AR 118584 A1 AR118584 A1 AR 118584A1
- Authority
- AR
- Argentina
- Prior art keywords
- raavs
- capsid proteins
- capsid
- proteins
- associated viruses
- Prior art date
Links
Landscapes
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
La presente se relaciona con virus adeno-asociados recombinantes (rAAVs) que poseen proteínas de la cápside diseñadas para incluir secuencias de aminoácidos que confieren y/o mejoran las propiedades deseadas. En particular, la presente proporciona proteínas de la cápside diseñadas que comprenden inserciones de péptidos a partir de proteínas heterólogas insertadas dentro o cerca de la región variable IV (VR-IV) de la cápside del virus, de modo tal que la inserción se encuentre expuesta en la superficie sobre la partícula de AVV. La presente también proporciona proteínas de la cápside que dirigen los rAAVs a los tejidos diana, en particular, proteínas de la cápside que comprenden péptidos derivados de eritropoyetina o dineína que están insertados dentro de las regiones variables expuestas en la superficie y que apuntan los rAAVs hacia el tejido retiniano y/o el tejido neural, incluyendo el sistema nervioso central, y suministran terapias para el tratamiento de los trastornos neurológicos y/u oculares.This relates to recombinant adeno-associated viruses (rAAVs) that possess capsid proteins designed to include amino acid sequences that confer and / or enhance the desired properties. In particular, the present provides designed capsid proteins that comprise peptide insertions from heterologous proteins inserted into or near variable region IV (VR-IV) of the virus capsid, such that the insert is exposed. on the surface on the AVV particle. The present also provides capsid proteins that direct rAAVs to target tissues, in particular capsid proteins comprising erythropoietin or dynein derived peptides that are inserted within surface exposed variable regions and that target rAAVs toward retinal tissue and / or neural tissue, including the central nervous system, and provide therapies for the treatment of neurological and / or ocular disorders.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962829608P | 2019-04-04 | 2019-04-04 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR118584A1 true AR118584A1 (en) | 2021-10-20 |
Family
ID=78803833
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP200100940A AR118584A1 (en) | 2019-04-04 | 2020-04-03 | RECOMBINANT ADENO-ASSOCIATED VIRUSES AND THEIR USES |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR118584A1 (en) |
-
2020
- 2020-04-03 AR ARP200100940A patent/AR118584A1/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| WO2020206189A9 (en) | Recombinant adeno-associated viruses and uses thereof | |
| MX2021014007A (en) | EpCAM BINDING PROTEINS AND METHODS OF USE. | |
| CR20180589A (en) | VARIANT CAPSULES OF ADENOASOCIATED VIRUSES AND METHODS OF USE OF THESE | |
| CL2020001360A1 (en) | Variant adeno-associated virus capsids and their use to inhibit angiogenesis. | |
| DOP2016000280A (en) | AAV VECTORS FOR GENETIC THERAPY OF THE RETINA AND THE CNS | |
| MX2022010175A (en) | Flt3 binding proteins and methods of use. | |
| PH12020500239A1 (en) | Factor viii (fviii) gene therapy methods | |
| EP4257155A3 (en) | Compositions and methods for treating wilson's disease | |
| WO2017187272A8 (en) | Optogenetic visual restoration using chrimson | |
| BR112022004027A2 (en) | Methods to treat ocular neovascular diseases using aav2 variants encoding aflibercept | |
| JP2017523239A5 (en) | ||
| WO2020219486A3 (en) | Collagen peptide-based medicament compositions and devices and methods of production and use thereof | |
| AR118584A1 (en) | RECOMBINANT ADENO-ASSOCIATED VIRUSES AND THEIR USES | |
| EA202192160A1 (en) | DELIVERY OF POLYNUCLEOTIDE CLN3 USING AN ADENO-ASSOCIATED VIRUS | |
| US20240050527A1 (en) | METHODS OF TREATING AGE-RELATED MACULAR DISEASES USING AIMP2-DX2 AND OPTIONALLY A TARGET SEQUENCE FOR miR-142 AND COMPOSITIONS THEREOF | |
| CN113584082B (en) | CRISPR/Cas9 gene editing system and application thereof in preparation of medicines for treating genetic sound nerve deafness | |
| Heo et al. | Intraperitoneal administration of adipose tissue‐derived stem cells for the rescue of retinal degeneration in a mouse model via indigenous CNTF up‐regulation by IL‐6 | |
| CN113181376A (en) | Application of Htra2 gene expression inhibitor in preventing acquired sensorineural deafness | |
| Kulchitsky et al. | Nasolacrimal way of stem cells implantation | |
| Zhang et al. | Involvement of upregulated P53-induced death domain protein in retinal ganglion cells apoptosis after optic nerve crush | |
| UY38881A (en) | METHODS FOR THE TREATMENT OF EYE NEOVASCULAR DISEASES USING AFLIBERCEPT VARIANTS OF AAV2 | |
| BR112022003206A2 (en) | METHODS TO TREAT NEOVASCULAR EYE DISEASES USING AAV2 VARIANTS ENCODING AFLIBERCEPTE | |
| AR118282A1 (en) | IMMUNOGENIC FORMULATIONS FOR THE TREATMENT OF CANCER | |
| Harvey | Gene therapy and the regeneration of retinal ganglion cell axons | |
| Saretzki | The wonderful new world of telomerase in the brain and its possible implications for neurodegenerative diseases |