Gene therapy for tuberous sclerosis
US-2024343768-A1 · Oct 17, 2024 · US
US10668129B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-10668129-B2 |
| Application number | US-201615576027-A |
| Country | US |
| Kind code | B2 |
| Filing date | May 20, 2016 |
| Priority date | May 21, 2015 |
| Publication date | Jun 2, 2020 |
| Grant date | Jun 2, 2020 |
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The present invention relates to the synergistic combination of the short and long Rod-Derived Cone Viability Factors and to methods for treating retinal degenerative diseases.
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The invention claimed is: 1. An expression vector comprising a first nucleic acid encoding a short isoform of the NXNL1 gene, Rod-derived Cone Viability Factor (RdCVF) and a second nucleic acid encoding a long isoform of the NXNL1 gene, RdCVFL, wherein said expression vector is an adeno-associated virus (AAV) and wherein said short isoform of RdCVF is the short isoform of human RdCVF and wherein said long isoform is the long isoform of human RdCVFL. 2. An expression vector according to claim 1 wherein said AAV is selected in the group consisting of AAV2, AAV9 and AAV7m8. 3. A method for treating a patient suffering from a retinal degenerative disease comprising the step of administering to said patient a therapeutically effective amount of an expression vector according to claim 1 . 4. A method according to claim 3 , wherein said retinal degenerative disease is retinitis pigmentosa. 5. A method according to claim 3 , wherein said expression vector is administered by intravenous injection or by intravitreal injection.
Demonstrated in vivo effect · CPC title
from mammals · CPC title
Ophthalmic agents · CPC title
from mammals · CPC title
Viral vectors · CPC title
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