Yin Linlin, Maddison Lisette A, Li Mingyu, Kara Nergis, LaFave Matthew C, Varshney Gaurav K, Burgess Shawn M, Patton James G, Chen Wenbiao
Department of Molecular Physiology and Biophysics, Vanderbilt University School of Medicine, Nashville, Tennessee 37232.
Department of Biological Science, Vanderbilt University, Nashville, Tennessee 37240.
Genetics. 2015 Jun;200(2):431-41. doi: 10.1534/genetics.115.176917. Epub 2015 Apr 8.
Determining the mechanism of gene function is greatly enhanced using conditional mutagenesis. However, generating engineered conditional alleles is inefficient and has only been widely used in mice. Importantly, multiplex conditional mutagenesis requires extensive breeding. Here we demonstrate a system for one-generation multiplex conditional mutagenesis in zebrafish (Danio rerio) using transgenic expression of both cas9 and multiple single guide RNAs (sgRNAs). We describe five distinct zebrafish U6 promoters for sgRNA expression and demonstrate efficient multiplex biallelic inactivation of tyrosinase and insulin receptor a and b, resulting in defects in pigmentation and glucose homeostasis. Furthermore, we demonstrate temporal and tissue-specific mutagenesis using transgenic expression of Cas9. Heat-shock-inducible expression of cas9 allows temporal control of tyr mutagenesis. Liver-specific expression of cas9 disrupts insulin receptor a and b, causing fasting hypoglycemia and postprandial hyperglycemia. We also show that delivery of sgRNAs targeting ascl1a into the eye leads to impaired damage-induced photoreceptor regeneration. Our findings suggest that CRISPR/Cas9-based conditional mutagenesis in zebrafish is not only feasible but rapid and straightforward.
利用条件性诱变可极大地促进基因功能机制的确定。然而,构建工程化条件性等位基因效率低下,且仅在小鼠中得到广泛应用。重要的是,多重条件性诱变需要大量的繁育工作。在此,我们展示了一种在斑马鱼(Danio rerio)中实现一代多重条件性诱变的系统,该系统利用了cas9和多个单向导RNA(sgRNA)的转基因表达。我们描述了用于sgRNA表达的5种不同的斑马鱼U6启动子,并证明了酪氨酸酶、胰岛素受体a和b的高效多重双等位基因失活,导致色素沉着和葡萄糖稳态方面的缺陷。此外,我们利用Cas9的转基因表达证明了时间和组织特异性诱变。cas9的热休克诱导表达可实现对tyr诱变的时间控制。cas9的肝脏特异性表达会破坏胰岛素受体a和b,导致空腹低血糖和餐后高血糖。我们还表明,将靶向ascl1a的sgRNA导入眼睛会导致损伤诱导的光感受器再生受损。我们的研究结果表明,基于CRISPR/Cas9的斑马鱼条件性诱变不仅可行,而且快速简便。