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Therapeutic compositions of mRNA and its use to treat diseases and disorders
专利权人:
Inc.;Translate Bio
发明人:
HEARTLEIN, Michael,HEARTLEIN MICHAEL
申请号:
ES14718277
公开号:
ES2692363T3
申请日:
2014.03.14
申请国别(地区):
ES
年份:
2018
代理人:
摘要:
A composition for use in a method of treating a subject who has a disease that is the result of a deficiency in a protein, wherein the composition comprises (a) at least one mRNA molecule, at least a portion of which encodes a fusion protein comprising a therapeutic protein and a polypeptide capable of binding to an Fc receptor; and (b) an administration vehicle, wherein the administration vehicle is polyethyleneimine (PEI) or a lipid nanoparticle, wherein the composition is administered to the subject by pulmonary delivery, and the disease: (a) is selected from a disorder of lysosomal storage, a metabolic disorder of the urea cycle, Pompe disease, Gaucher disease, beta-thalassemia, Huntington's disease; Parkinson's disease; muscular dystrophies (such as Duchenne and Becker); hemophilia diseases (such as hemophilia B, hemophilia A, SMN1-related spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), GALT-related galactosemia, SLC3A1-related disorders including cystinuria, COL4A5-related disorders including syndrome of Alport deficiencies of galactocerebrosidase, adrenoleukodystrophy and X-linked adrenomyeloneuropathy; Friedreich ataxia; Pelizaeus-Merzbacher disease; tuberous sclerosis related to TSC1 and TSC2; Sanfilippo bB syndrome (MPS IIIB); CTR1-related cystinosis; including fragile X syndrome, fragile X tremor / ataxia syndrome and fragile X premature ovarian insufficiency syndrome; Prader-Willi syndrome; hereditary hemorrhagic telangiectasia (AT); Niemann-Pick Type C1 disease; lipofuscinosis-related diseases neuronal ceroids including neuro zero-lipofuscinosis juvenile nal (JNCL), juvenile Batten disease, Santavuori-Haltia disease, Jansky-Bielschowsky disease and PTT-1 and TPP1 deficiencies; childhood ataxia related to EIF2B1, EIF2B2, EIF2B3, EIF2B4 and EIF2B5 with hypomyelination / disappearance of white matter from the central nervous system; Type 2 episodic ataxia related to CACNA1A and CACNB4; MECP2-related disorders, including classical Rett s
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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