The compound suppresses lipogenesis in a concentration-dependent manner, with doses of 30-60 M reducing lipid accumulation by 50-70% in cultured adipocytes
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For example, in certain embodiments, a sustained and/or extended release formulation described herein comprises a combination of poloxamer 407 (pluronic F127) and carboxymethylcellulose (CMC) in a ratio of about 40:1, about 35:1, about 30:1, about 25:1, about 20:1, about 15:1, about 10:1, or about 5:1.4%, about 4.5%, or about 5% of the total weight of the formulation or composition
For example, in a mouse model of mucopolysaccharidosis (MPS VII), transgene delivery of b-glucuronidase in neonatal mice using an AAV2 vector resulted in the mice developing hepatocellular carcinoma (HCC)with evidence of AAV integration within the tumors [149, 150]