US Patent Issued to The Children's Hospital of Philadelphia on Nov. 3 for "Combined transgene and intron-derived miRNA therapy for treatment of SCA1" (Pennsylvania Inventors)

ALEXANDRIA, Va., Nov. 3 -- United States Patent no. 12,778,977, issued on Nov. 3, was assigned to The Children's Hospital of Philadelphia (Philadelphia).

"Combined transgene and intron-derived miRNA therapy for treatment of SCA1" was invented by Beverly L. Davidson (Philadelphia), Ellie Carrell (Philadelphia), Alejandro Mas Monteys (Philadelphia) and Megan S. Keiser (Philadelphia).

According to the abstract* released by the U.S. Patent & Trademark Office: "Provided herein are nucleic acids that comprise both an expression cassette for a therapeutic protein (e.g., Ataxin-1-like) and an expression cassette for a therapeutic inhibitory RNA (e.g., a miRNA that targets ataxin-1 mRNA). In some instances, the expression cassette for the therapeu...