SLC13A5 GENE THERAPY VECTORS AND USES THEREOF
Abstract:
The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SLC13A5 loss, misfunction and/or deficiency, including neurological disorders, diseases, and conditions such as epileptic encephalopathy. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for an SLC13A5 polypeptide.
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