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Methods and compositions for Intrathecal Administration of n-acetyl glucosaminidasa
专利权人:
SHIRE HUMAN GENETIC THERAPIES; INC.
发明人:
申请号:
ARP110102244
公开号:
AR081680A1
申请日:
2011.06.27
申请国别(地区):
AR
年份:
2012
代理人:
摘要:
A n-acetylglucose microsa. Requiremen1: a method for the treatment of B-type (sanb) triphenol syndrome;This includes the steps of providing the recombinant protein n-acetylglucose micro (NAGLU) from the inside to those in need of treatment. Claim 2: the method of claim 1, wherein the recombinant NAGLU protein is a molten protein, including a NAGLU domain and a dissolved target segment. Claim 29: the therapeutic combining protein comprises a NAGLU domain: a target, i.e. a target, once managed, the therapeutic combining protein is targeted at lisosoras and is active in vivo in treatment. Claim 31: therapeutic combined protein of claim 29,NAGLU rule consists of a series of amino acids, identical to SEQ ID 1 (mature human protein). Claim 32: therapeutic combined proteins in any 29 to 31 claims, the target of which is IGF LL. Claim 34: therapeutic combined protein of claim 33, in which IGF-II comprises a series of amino acids, including residues 8-67 (SEQ ID No.2) caused by second global warming in mature humans. Claim 35: any 29 to 34 claims for therapeutic concomitant proteins,The fusion protein also includes a target segment connecting the NAGLU domain and the troposphere. Claim 36: the therapeutic combined protein of claim 35, wherein the attachment comprises a series of amino acids, consisting of gapggagaggagaggagaggagaggagaggap (SEQ ID No.4). Claim 39: an internal tube system, comprising a liquid access device, a hollow body with a first fluid communication port and a second fixed fluid port to be inserted into the spinal cord;There is also a fixation device to determine the hollow body inserted into the spinal cord.Formulaciones estables para la administración por vía IT de a N-acetilglucosaminidasa.Reivindicación 1: Un método para tratar del síndrome de Sanfilippo tipo B (San B), que comprende un paso de administrar por vía intratecal una proteína a-N-acetilglucosaminidasa (Naglu) recombinante a un sujeto que necesita recibir tratamiento. Reivindicación 2: El m
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