Kiyoshi TACHIKAWA,Carlos Gustavo PEREZ-GARCIA,Padmanabh CHIVUKULA,Hari Prakash BHASKARAN,Christian W. COBAUGH,Sean Christopher DAUGHERTY
申请号：
US16617431
公开号：
US20200149017A1
申请日:
2018.05.31
申请国别(地区):
US
年份:
2020
代理人:
摘要：
This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL), or a fragment thereof having AGL activity. The polynucleotide and oligomer molecules are expressible to provide the human AGL or a fragment thereof having AGL activity. The molecules can be used as active agents to express an active polypeptide or protein in cells or subjects. The agents can be used in methods for ameliorating, preventing, delaying onset, or treating a disease or condition associated with reduced activity of amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL) in a subject.
