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Design of a Dendrimeric Nanomedicine Platform for the Treatment of a Neuro-Degenerative Disease, X-ALD

机译:用于治疗神经退行性疾病,X-ALD的树枝状纳米药物平台的设计

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PAMAM-drug (D-PBA) conjugates were successfully synthesized with high drug payload and characterized. The sustained and slow drug release behaviour was verified in in vitro conditions such as PBS (with and without enzyme) and plasma. Later, these conjugates were evaluated for their ability to decrease VLCFA in a mouse model of A LD. It was demonstrated that this drug-conjugated dendrimers provides slow and sustained drug release which reduced VLCFA in vivo and may be evaluated as apotential nanomedicine for the prolonged treatment of X-linked ALD.
机译:PAMAM-药物(D-PBA)偶联物已成功合成,具有较高的药物有效负载并进行了表征。在体外条件下,例如PBS(有或没有酶)和血浆中,验证了药物的持续释放和缓慢释放行为。随后,评估这些缀合物在A LD小鼠模型中降低VLCFA的能力。已证明该药物缀合的树状聚合物提供缓慢且持续的药物释放,其在体内降低了VLCFA,并且可以被认为是潜在的纳米药物,用于延长治疗X-连锁的ALD。

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