公开(公告)号：US20230089490A1

公开(公告)日：2023-03-23

申请号：US17767727

申请日：2020-10-09

Applicant: University of Massachusetts

Inventor： Guangping Gao ,  Craig Mendonca ,  Dan Wang

IPC: C12N15/113 ,  C12N15/86 ,  A61K48/00 ,  A61P7/00 ,  A61P11/00 ,  A61P21/00

Abstract: Aspects of the disclosure relate to compositions and methods for treating certain diseases associated with the presence of one or more premature stop codons in a gene, for example dominantly inherited diseases or recessively inherited diseases. In some embodiments, compositions comprise a vector (e.g., a viral vector, such as an rAAV vector) encoding one or more synthetic suppressor transfer RNAs (tRNAs) configured to read-through certain stop codons (e.g., premature stop codons). In some embodiments, the disclosure relates to methods for treating Hurler syndrome comprising administering such vectors to a subject.