TREATMENT OF SANFILIPPO SYNDROME TYPE B

Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a sui...

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Main Authors HUANG, Yan, STRACK-LOGUE, Bettina, HOLMES, Kevin, LUNDBERG, Dianna, PFEIFER, Richard, CONCINO, Michael, F, PAN, Jing, NORTON, Angela, ISKENDERIAN, Andrea, MARTINI, Paolo, CALIAS, Pericles, ZHANG, Bohong, MEIYAPPAN, Muthuraman, ROMASHKO, Alla, ALESSANDRINI, Mary
Format Patent
LanguageEnglish
French
German
Published 09.08.2017
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:Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
Bibliography:Application Number: EP20110799039