Molecular Therapy of Melanocortin-4-Receptor Obesity by an Autoregulatory BDNF Vector

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Molecular Therapy of Melanocortin-4-Receptor Obesity by an Autoregulatory BDNF Vector

Mutations in the melanocortin-4-receptor (MC4R) comprise the most common monogenic form of severe early-onset obesity, and conventional treatments are either ineffective long-term or contraindicated. Immediately downstream of MC4R-in the pathway for regulating energy balance-is brain-derived neurotrophic factor (BDNF). Our previous studies show that adeno-associated virus (AAV)-mediated hypotha...

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Melanocortin 2 receptor (MC2R) is the only canonical ACTH receptor. Its functional expression requires the presence of an accessory protein, known as melanocortin receptor 2 accessory protein 1 (MRAP1). The vertebrate genome exhibits a paralogue gene called MRAP2, which is duplicated in zebrafish (MRAP2a and MRAP2b), although its function remains unknown. In this paper, we demonstrate that MRAP...

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ژورنال

عنوان ژورنال: Molecular Therapy - Methods & Clinical Development

سال: 2017

ISSN: 2329-0501

DOI: 10.1016/j.omtm.2017.09.005