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US 2017/0246263 A1Published application
Lysosomal targeting and uses thereof
AssigneeShire Human Genetic Therapies, Inc.
InventorsMichael F. CONCINO, Bettina STRACK-LOGUE, Muthuraman MEIYAPPAN, Angela W. NORTON, Bohong ZHANG, Andrea ISKENDERIAN, Jianwen FENG, Kevin HOLMES, Pan Jing
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Abstract
The invention provides compositions and methods for effective lysosomal targeting mediated by SORT1. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Sanfilippo syndrome type B.
Claims27
A targeted therapeutic comprising:
- a lysosomal enzyme;
- and
- a lysosomal targeting moiety that binds to SORT1.
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Dependencies are inferred from the claim text.