COMPOSITIONS AND METHODS FOR TREATMENT OF HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS

摘要

Provided herein are nucleic acids, vectors, and cells containing an expression-optimized codon that encodes a Munc13-4 polypeptide or a STXBP2 polypeptide. Also provided are methods of making and using the nucleic acids, vectors, and cells. Also provided herein are methods of treating of Hemophagocytic Lymphohistiocytosis (HLH) in a subject.