LYSOSOMAL TARGETING AND USES THEREOF

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.

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Bibliographic Details
Main Authors NORTON, Angela W, CONCINO, Michael F, STRACK-LOGUE, Bettina, ISKENDERIAN, Andrea, HOLMES, Kevin, ZHANG, Bohong, MEIYAPPAN, Muthuraman, FENG, Jianwen, PAN, Jing
Format Patent
LanguageEnglish
French
German
Published 26.07.2017
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Summary: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.
Bibliography:Application Number: EP20150756512