Constructs comprising neuronal viability factors and uses thereof
AssigneesSparingvision, Inserm (Institut National De La Sante Et De La Recherche Medicale), Sorbonne Universite
InventorsThierry LEVEILLARD, Najate AÏT-ALI MAAMRI, Fréderic BLOND, José-Alain SAHEL, Géraldine PUEL, Emmanuelle CLERIN
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Abstract
The present invention relates to improved constructs comprising the short and long Rod-Derived Cone Viability Factors and to methods for treating retinal degenerative diseases.
Claims20
An adeno-associated vector (AAV) comprising:
- a first expression cassette comprising a first nucleic acid encoding RdCVF and
- a second expression cassette comprising a second nucleic acid encoding RdCVFL, wherein said first and second expression cassettes display less than 200 contiguous identical nucleotides.
Method for treating a patient suffering from a retinal degenerative disease comprising the step consisting of administering to said patient a therapeutically effective amount of an adeno-associated vector (AAV) comprising:
- a first expression cassette comprising a first nucleic acid encoding RdCVF and
- a second expression cassette comprising a second nucleic acid encoding RdCVFL,
- wherein said first and second expression cassettes display less than 200 contiguous identical nucleotides.
An AAV comprising a nucleic acid having the sequence set forth in SEQ ID NO:10, wherein said nucleic acid having the sequence as set forth in SEQ ID NO:10 is not present in an expression cassette.
Dependencies are inferred from the claim text.