公开(公告)号：US20210222141A1

公开(公告)日：2021-07-22

申请号：US17055523

申请日：2019-05-15

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Xavier ANGUELA ,  Sean ARMOUR ,  Jayme NORDIN

IPC: C12N9/26 ,  C12N15/86 ,  A61K48/00

Abstract: The invention provides nucleic acids encoding acid a-glucosidase (GAA). In certain embodiments, nucleic acids have greater than about 86% sequence identity to a sequence selected from the group consisting of any of the sequences set forth as SEQ ID NOs:1-5. In certain embodiments, nucleic acids encoding acid a-glucosidase (GAA) contain less than 127 CpG dinucleotides. Expression cassettes, vectors, cells and cell lines and methods of using such nucleic acids encoding acid a-glucosidase (GAA) are also provided.