US Patent Issued to University of Massachusetts on Nov. 25 for "Gene therapy for spinal muscular atrophy" (Massachusetts Inventors)

ALEXANDRIA, Va., Nov. 25 -- United States Patent no. 12,478,692, issued on Nov. 25, was assigned to University of Massachusetts (Westborough, Mass.).

"Gene therapy for spinal muscular atrophy" was invented by Jun Xie (Worcester, Mass.), Guangping Gao (Worcester, Mass.), Qing Xie (Worcester, Mass.) and Hong Ma (Worcester, Mass.).

According to the abstract* released by the U.S. Patent & Trademark Office: "Aspects of the disclosure relate to compositions and methods for treating spinal muscular atrophy (SMA). The disclosure is based, in part, on isolated nucleic acids and vectors (e.g., viral vectors, such as rAAV vectors) encoding SMN1. In some embodiments, the expression of SMN1 is driven by a native SMN1 promoter or a variant thereof. In ...