Adeno-Associated Viral (AAV)-Mediated Sgpl1 Gene Therapy For Treatment Of Sphingosine-1-Phosphate Lyase Insufficiency Syndrome (SPLIS)
OwnerJulie D. Saba, Piming Zhaoinventors
Life of this patent
Estimating the term…
Abstract
The present disclosure provides methods and compositions for transferring a gene encoding sphingosine-1-phosphate lyase (SPL) in a subject in need thereof by using recombinant AAV virions. The subject may have or may develop SPL insufficiency syndrome (SPLIS). The subject may be identified on the basis of a genetic test and/or SPLIS symptoms. The methods and compositions are also useful in lowering circulating sphingosine-1-phosphate levels in a subject having elevated S1P levels.
Claims39
A method for treating SPL insufficiency syndrome (SPLIS) in a subject, the method comprising administering to the subject a therapeutically effective dose of a recombinant adeno-associated viral (rAAV) virion comprising a nucleic acid encoding sphingosine-1-phosphate lyase (SPL), wherein administering the rAAV virion results in expression of the SPL in the subject thereby treating the SPLIS in the subject.
A method for preventing a subject from developing SPL insufficiency syndrome (SPLIS), the method comprising administering to the subject a therapeutically effective dose of a recombinant adeno-associated viral (rAAV) virion comprising a nucleic acid encoding sphingosine-1-phosphate lyase (SPL), wherein administering the rAAV virion results in expression of the SPL in the subject thereby preventing the subject from developing SPLIS.
A method for reducing circulating sphingosine-1-phosphate (S1P) level in a subject having increased circulating S1P level, the method comprising administering to the subject a therapeutically effective dose of a recombinant adeno-associated viral (rAAV) virion comprising a nucleic acid encoding sphingosine-1-phosphate lyase (SPL), wherein administering the rAAV virion results in expression of SPL in the subject thereby reducing the circulating S1P level in the subject.
A recombinant Adeno-associated viral (rAAV) vector comprising an AAV inverted terminal repeat, a promoter/enhancer, a nucleic acid sequence encoding sphingosine-1-phosphate lyase (SPL), and an AAV inverted terminal repeat.
A recombinant adeno-associated viral (rAAV) virion comprising a nucleic acid encoding sphingosine-1-phosphate lyase (SPL).
Dependencies are inferred from the claim text.