多肽分子RC-100 有望成为新一代基因治疗工具
生物谷援引华文生技网报道：基因治疗的过程中，最大的瓶颈，在于如何的把特定的基因，送到特定的组织细胞中，虽然科学家利用病毒的基因片段，作为运送治疗用基因的载体，但实际上运作的效率并不高，最近由加州大学洛杉矶分校的科学家，发表在最新一期 AIDS研究与人类逆转病毒研究 (AIDS Research and Human Retroviruses)上文章，声称发现了一个特殊的多肽分子RC-100 (Retrocyclin-1) 的环状多肽，很可能成为下一代基因治疗的工具。
在于研究抗HIV入侵的机制，结果研究人员发现一个称为RC-100 (Retrocyclin-1) 的环状多肽，可以阻挡HIV功击CD4免疫细胞的机会，而另外一个称为 RC-111的分子也是一个环状多肽，不过却有着截然不同的功能, 它可以增加 HIV病毒攻击宿主细胞的能力，这两个分子的骨架，非常类似于 18个氨基酸的基团，而且两分子的氨基酸序列，正好相反。进一步研究发现，RC-100约可以抑制 95%HIV-1病毒对CD4 细胞的攻击，而RC-111 却增加了病毒5 倍的攻击力。
UCLA 的研究人员表示，虽然目前并不十分清楚这段特殊序列背后所隐藏的机制，但从基因载体的角度看来，科学家可以说找到了一个更有效率的基因治疗工具。
(资料来源 : biocompare)
英文原文链接：
http://news.biocompare.com/newsstory.asp?id=182357
原始出处:
Retrocyclin-1 (RC-100) is a cyclic octadecapeptide whose primary structure is based on the sequence of an expressed human θ-defensin pseudogene. RC-111 has the same amino acid sequence as RC-100 and is also cyclic, but its residues are placed in reverse order along the peptide's backbone. We quantified the effects of RC-100 and RC-111 on HIV-1 infection using HIV clones that expressed green fluorescent protein. Whereas 0.2 μg/ml of RC-100 inhibited infection of CD4-positive cells by approximately 80%, its retroanalogue significantly enhanced infection of the cells. RC-100 and RC-111 also demonstrate their effects in HIV infection of CD4-negative cells. Whereas 40 ng/ml of RC-111 significantly enhanced infection of CD4-negative cells by HIV-1, RC-100 demonstrated significant inhibition of HIV infection with a concentration of approximately 10 μg/ml. RC-111ox, an acyclic variant of RC-111 with a β-hairpin structure, also enhanced HIV-1 infection, but did so less effectively than cyclic RC-111. The divergent actions of RC-100 and RC-111 show that topology and polarity of θ-defensin peptides can determine their effect on HIV infection. The ability of RC-111 to enhance HIV-1 infection might prove useful in developing peptides that can enhance gene delivery by HIV-based lentiviral vectors.
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