ADENO-ASSOCIATED VIRAL (AAV)-MEDIATED SGPL1 GENE THERAPY FOR TREATMENT OF SPHINGOSINE-1-PHOSPHATE LYASE INSUFFICIENCY SYNDROME (SPLIS)

摘要

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.