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The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma iduronidase (IDUA) enzyme activity of ascending doses of SB-318. SB-318 is an intravenously delivered ZFP Therapeutic for genome editing. It inserts a correct copy of the IDUA gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDUA enzyme.
The objectives of the study are to provide long term expression of IDUA and improve the current clinical outcome of enzyme replacement therapy (ERT) or hematopoietic stem cell transplantation (HSCT) therapy in subjects with MPS I, a recessive lysosomal storage disorder that results from mutations in the gene encoding IDUA. Placement of the corrective copy of IDUA transgene into the genome of the subject's own hepatocytes under the control of the highly expressed endogenous albumin locus, is expected to provide permanent, liver-specific expression of iduronidase for the lifetime of an MPS I patient.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
University of Minnesota
Not yet recruiting
Published on BioPortfolio: 2016-03-09T03:08:23-0500
Arnica montana is a homeopathic drug that is made in accordance with the official methods of the Homeopathic Pharmacopoeia of the United States (HPUS) and is commonly used by cosmetic surg...
Enlarged tonsils and malocclusion has relationship with sleep disturbance in children. The consequences of these features can include aggravation of craniofacial growth such that the devel...
The purpose of this study is to assess the effects of repeated RIPC and exercise, on exercise performance, skeletal muscle responses and circulating cellular and humoral biology in humans
The substitution of raltegravir for the NRTIs will result in some reversal of the long term adverse effect of lipodystrophy (specifically peripheral lipoatrophy) that is associated with th...
Brief Summary: RATIONALE: Comparing results of diagnostic procedures, such as esophagoscopy, done before and after radiation therapy and/or chemotherapy may help doctors predict a patient...
To conduct a proof-of-concept pilot evaluation of the self-directed format of Walk With Ease (WWE), a 6-week walking program developed for adults with arthritis, in patients with systemic lupus erythe...
Headaches are common in patients with systemic lupus erythematosus (SLE). It is important to identify the exact cause of headaches in SLE to avoid unnecessary steroid or immunosuppressive therapy like...
An ionophorous, polyether antibiotic from Streptomyces chartreusensis. It binds and transports cations across membranes and uncouples oxidative phosphorylation while inhibiting ATPase of rat liver mitochondria. The substance is used mostly as a biochemical tool to study the role of divalent cations in various biological systems.
An organothiophosphate insecticide.
Places where animals are slaughtered and dressed for market.
Shortened forms of written words or phrases used for brevity.
That portion of the body that lies between the THORAX and the PELVIS.
Blood is a specialized bodily fluid that delivers necessary substances to the body's cells (in animals) – such as nutrients and oxygen – and transports waste products away from those same cells. In vertebrates, it is composed of blo...
Enzymes are proteins that catalyze (i.e., increase the rates of) chemical reactions. In enzymatic reactions, the molecules at the beginning of the process, called substrates, are converted into different molecules, called products. Almost all chemical re...
Bioinformatics is the application of computer software and hardware to the management of biological data to create useful information. Computers are used to gather, store, analyze and integrate biological and genetic information which can then be applied...