重组杆状病毒作为一种新型的基因治疗载体的研究
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摘要
长期以来,由于其宿主特异性,杆状病毒载体仅限于在昆虫细胞内表达外源蛋白。但最近的研究发现,携带哺乳动物启动子的重组杆状病毒能有效转导哺乳动物细胞并且可以在哺乳动物细胞中高效表达外源蛋白。由于其转导效率高、易操作、增殖滴度高、易扩增、载体容载量大、在哺乳动物细胞中不复制和生物安全性好等优点,重组杆状病毒有望成为良好的基因治疗载体。
Apoptin是一种来源于鸡贫血病毒的小蛋白,能特异性诱导大量的肿瘤细胞和转化细胞产生凋亡,而对正常二倍体细胞无凋亡诱导作用。特异的肿瘤细胞杀伤性使Apoptin成为癌症基因治疗的一种新的工具。
Duchenne型肌营养不良症(Duchenne musculardystrophy,DMD)是一种以骨骼肌进行性变性、坏死为主要病理特征的致死性X-性连锁隐性遗传性肌病,目前尚无有效的治疗方法,基因治疗被认为是治疗该病最有希望的途径。但是现有的基因治疗载体的载体容载量少、需要复制辅助病毒和存在着生物安全性等局限性大大的限制它的临床应用。因此提高治疗基因在体内的表达效率以及选择合适的载体将是治疗DMD获得成功的关键。
鉴于上述的研究背景,本研究探讨了含有Apoptin蛋白的重组杆状病毒在体外、体内的抑瘤的作用;研究了含有microdystrophin蛋白的重组杆状病毒在体内和体外的microdystrophin表达情况,比较了重组杆状病毒和重组腺病毒在体内基因治疗mdx鼠的效果。主要研究内容如下:
1.重组杆状病毒BV-Apoptin的构建
从质粒pVAX-Apoptin中PCR扩增全长的Apoptin片段,经酶切克隆到真核表达载体pcDNA3.1-HisB中,构建重组质粒pcDNA3.1-His-Apoptin。然后将CMV启动子驱动的编码Apoptin蛋白的整个读码框插入转移载体pFast-VSV-G中VSV-G的下游,构建转移载体pFast-G-Apoptin。将此重组质粒转化DH_(10)Bac大肠杆菌,通过蓝白斑菌落及抗性筛选,获得重组穿梭载体Bacmid-Apoptin,提取其基因组并转染sf9昆虫细胞,获得重组杆状病毒BV-Apoptin。
2.重组杆状病毒BV-Apoptin的体外转导研究
重组杆状病毒BV-Apoptin在细胞中有正确的定位。在人肝癌细胞HepG2中Apoptin主要定位于细胞核,在人胚肾细胞HEK-293中,Apoptin定位于细胞质中。体外转导试验表明,构建的重组杆状病毒BV-Apoptin能高效转导HepG2细胞,Apoptin的表达水平与杆状病毒的感染剂量成正相关,当感染复数越高,表达Apoptin蛋白的细胞数越多。
3.重组杆状病毒BV-Apoptin在体外、体内的抑瘤作用
将重组杆状病毒BV-Apoptin分别以不同感染复数转导HepG2细胞,通过DNA-Ladder和TUNEL检测细胞凋亡。结果显示,重组杆状病毒BV-Apoptin能够诱导转导的人肝癌细胞HepG2的产生凋亡,并且BV-Apoptin诱导肿瘤细胞凋亡的能力呈剂量依赖性。实验利用异体同源肿瘤细胞系移植的方法复制了C57BL/6小鼠荷H22肿瘤模型,研究构建的重组杆状病毒BV-Apoptin的体内抑瘤效果。结果显示,注射BV-Apoptin能够明显抑制小鼠肿瘤的生长,同时也提高荷瘤小鼠的存活率。以上结果提示,重组杆状病毒BV-Apoptin有望成为治疗肿瘤的一种新的有效手段。
4.重组杆状病毒BV-MICDYS的构建
从载体pcDNA3.1(+)中酶切CMV启动子的表达框,插入转移载体pFast-VSV-G中VSV-G的下游,构建转移载体pFast-G-CMV;然后从质粒pBSK-MICRO酶切全长的microdystrophin基因,分别克隆到pFast-G-CMV中,构建质粒pFast-G-MICDYS。转化DH_(10)Bac大肠杆菌,通过蓝白斑菌落及抗性筛选,获得重组穿梭载体Bacmid-MICDYS,提取其基因组并转染sf9昆虫细胞,获得重组杆状病毒BV-MICDYS。
5.重组杆状病毒BV-MICDYS转导C2C12细胞
将构建的重组杆状病毒BV-MICDYS以不同的感染复数(50、100、200MOI)转导C2C12细胞,转导后48h检测microdystrophin基因的表达。结果显示,重组杆状病毒BV-MICDYS能高效转导C2C12细胞,呈现明显的剂量依赖性。结果还显示,相同感染复数的Ad-MICDYS感染的C2C12细胞中microdystrophin蛋白表达数量低于相同感染复数BV-MICDYS转导的C2C12细胞中microdystrophin蛋白表达数量,说明了在C2C12细胞中,杆状病毒有着比腺病毒更高的靶基因表达水平的能力。
6.体内研究重组杆状病毒BV-MICDYS在mdx TA中的转导
将重组杆状病毒BV-MICDYS分别以不同的剂量(10~(10)pfu/mL,10~9pfu/mL,10~8pfu/mL)注射到mdx TA,2周和8周后做肌肉组织冰冻切片,HE检测肌肉病理的改善情况,免疫组化检测microdystrophin蛋白的表达,同时做原位检测等长拉伸诱导损伤后TA肌肉收缩力恢复情况。试验结果表明,在注射后2周,重组杆状病毒BV-MICDYS能介导microdystrophin蛋白在小鼠体内的肌肉细胞中高效表达,并且表达水平其呈现剂量依赖性,能更好的改善肌肉病理状况。但重组杆状病毒在注射后8周,未检测到其治疗效果,显示重组杆状病毒BV-MICDYS较适用于DMD的短期治疗。
The baculovirus Autographa californica multiple Nucleopolyhedrovirus (AcMNPV) has been traditionally used as an efficient vehicle to over-express recombinant proteins in insect cells. Its host specificity was originally thought to be restricted to cells derived from arthropods. Recently, however, accumulating evidence has revealed that baculovirus, carrying mammalian cell-active promoters, is capable of transferring and effective expressing foreign genes in a variety of mammalian cells. Thanks to its highly efficient gene delivery mechanism, increasing interest has been shown in baculovirus as a novel vector for gene therapy. In addition, other potential advantages, including easy manipulation, high recombinant viral titers, simple scale-up, a large DNA insertion capacity, lack of replication in mammalian cells, and lack of toxicity, make this vector highly promising for gene therapy.
Apoptin, a protein derived from the chicken anemia virus, can induce programmed cell death in a large panel of human transformed and malignant cells, but not in normal, diploid cells. The intrinsic tumor specificity of Apoptin makes it a promising new tool for cancer gene therapy.
Duchenne musclar dystrophy (DMD) is an X-linked recessive disease with characteristic of progressive degeneration and necrosis in skeletal muscle. There is no effective treatment for DMD at present, gene therapy is a promising therapeutical approaches for the treatment of DMD. Howeve, DMD gene therapy is challenged by the viral vectors for their small insert capacity, replication-deficient and require helper virus functions for propagation and raising biosafety concerns. These all impede their clinical application. Enhancement the experssed efficiency in vivo and selection ideal vector are the keys to success to treat DMD.
Based on the above mentioned research background, the present study investigate the anti-tumor effects in vitro and in vivo of a recombinant baculovirus encoding Apoptin protein, evaluated the microdystrophin protein expression of the recombinant baculovirus containing microdystrophin gene, at last, compared the gene therapy ability of the recombinant baculovirus and recombinant adenovirus in mdx mice. The main projects are:
1. Construction of recombinant baculovirus expressing the Apoptin protein
Full-length cDNA for Apoptin was amplified by polymerase chain reaction from plasmid pVAX-Apoptin, and subcloned into pcDNA-HisB vector to generate pcDNA3.1-His-Apoptin. The DNA fragment containing CMV-IE enhancer/promoter-Apoptin was obtained from pc-Apoptin and inserted into pFast-VSV-G to generate pFB-G-Apoptin. The recombinant baculoviruses were subsequently generated by using the Bac-to-Bac(?) system (Invitrogen) following the manufacturer's instructions. The virus was further amplified by propagation in Sf-9 cells.
2. The research on effect of transduction with recombinant baculovirus BV-Apoptin
We investigate the correct subcellular localization of BV-Apoptin in cells. We found that Apoptin was mainly located in the nucleus of HepG2 cells, whereas in normal HEK293 cells BV-Apoptin was localized in the cytoplasm. The results indicated that BV-Apoptin could mediate efficient gene delivery and the greater gene transduction efficiency of the BV-Apoptin was confirmed by comparing the Apoptin expression level in a variety of multiplity of infection.
3. The antitumor effect of BV-Apoptin in vitro and in vivo
To determine whether BV-Apoptin can induce apoptosis in transduced HepG2 cells, the DNA ladder and TUNEL assays were performed. An obvious DNA ladder pattern could be observed in BV-Apoptin-transduced HepG2 cells. The results indicated that BV-Apoptin induced apoptosis in a dose-dependent manner, specifically in transduced tumor cells but not in transduced normal cells. C57BL/6 mice model bearing H22 hepatoma was constructed by transplanting H22 cells into the right hind limb of the mice and the anti-tumor effect on H22 hepatoma of BV-Apoptin was observed. The results indicated that the treatment with BV-Apoptin can confer significant survival benefits and reduction of tumor size in vivo. Taken altogether, BV-Apoptin shows the good anti-tumor effect may be used as new and powerful strategy for cancer bio-therapy in future.
4. Construction of recombinant baculovirus expressing the microdystrophin protein
The DNA fragment containing CMV-IE enhancer/promoter was released from pcDNA3.1 (+) and inserted into pFast-VSV-G, resulting in pFB-G-CMV. Full-length cDNA for human microdystrophin gene was acqured from the plasmid pBSK-MICRO and subcloned into pFB-G-CMV to generate pFB-G-MICDYS. The recombinant baculoviruses were subsequently generated by using the Bac-to-Bac(?) system (Invitrogen) following the manufacturer's instructions. The virus was further amplified by propagation in Sf-9 cells.
5. Research on the effect of transduction with BV-MICDYS in C2C12 cells
The C2C12 cells were transduced with BV-Apoptin at different MOIs (50, 100 and 200). At 48 h after infection, the expression of microdystrophin was detected. Obvious differences in number of positive fluorescence cells were observed between wells infected with 50,100, 200 MOI of BV-MICDYS as the progression of the transduction. The microdystrophin protein expression by BV-MICDYS is in a dose-dependent manner in C2C12 cells. The results also indicated that in the same dosage, the microdystrophin positive of BV-MICDYS is higher than the microdystrophin expression with recombinant adenovirus Ad-MICDYS. These results indicated that BV-MICDYS could mediate efficient gene delivery and expression of microdystrophin protein in a dose-dependent manner in mammalian cells, and the transduction efficiency was higher than adenovirus.
6. Research on the effect of transduction with recombinant baculovirus mediated microdystrophin in mdx mice
TA muscles of mdx mice were injected with 10~(10)pfu/mL, 10~9pfu/mL, 10~8pfu/mL of recombinant baculovirus BV-MICDYS. Cryostat sections were prepared from the snap frozen muscle samples after 2 weeks and 8 weeks injection. HE evaluated pathological change of TA and immunocytochemistry evaluated the expression of microdystrophin; at the same time, the isometric contractile properties of TA were tested in situ. The results indicated in 2 weeks that transduction of the microdystrophin BV-MICDYS could retard significantly the degeneration and distribution in TA of mdx mice. The gene therapy ability of BV-MICDYS is in a dose-dependent pattern. But the BV-MICDYS lost its ability in gene therapy ability 8 weeks after injection. These results indicated that BV-MICDYS are suit for DMD short-term gene therapy.
Apoptin是一种来源于鸡贫血病毒的小蛋白,能特异性诱导大量的肿瘤细胞和转化细胞产生凋亡,而对正常二倍体细胞无凋亡诱导作用。特异的肿瘤细胞杀伤性使Apoptin成为癌症基因治疗的一种新的工具。
Duchenne型肌营养不良症(Duchenne musculardystrophy,DMD)是一种以骨骼肌进行性变性、坏死为主要病理特征的致死性X-性连锁隐性遗传性肌病,目前尚无有效的治疗方法,基因治疗被认为是治疗该病最有希望的途径。但是现有的基因治疗载体的载体容载量少、需要复制辅助病毒和存在着生物安全性等局限性大大的限制它的临床应用。因此提高治疗基因在体内的表达效率以及选择合适的载体将是治疗DMD获得成功的关键。
鉴于上述的研究背景,本研究探讨了含有Apoptin蛋白的重组杆状病毒在体外、体内的抑瘤的作用;研究了含有microdystrophin蛋白的重组杆状病毒在体内和体外的microdystrophin表达情况,比较了重组杆状病毒和重组腺病毒在体内基因治疗mdx鼠的效果。主要研究内容如下:
1.重组杆状病毒BV-Apoptin的构建
从质粒pVAX-Apoptin中PCR扩增全长的Apoptin片段,经酶切克隆到真核表达载体pcDNA3.1-HisB中,构建重组质粒pcDNA3.1-His-Apoptin。然后将CMV启动子驱动的编码Apoptin蛋白的整个读码框插入转移载体pFast-VSV-G中VSV-G的下游,构建转移载体pFast-G-Apoptin。将此重组质粒转化DH_(10)Bac大肠杆菌,通过蓝白斑菌落及抗性筛选,获得重组穿梭载体Bacmid-Apoptin,提取其基因组并转染sf9昆虫细胞,获得重组杆状病毒BV-Apoptin。
2.重组杆状病毒BV-Apoptin的体外转导研究
重组杆状病毒BV-Apoptin在细胞中有正确的定位。在人肝癌细胞HepG2中Apoptin主要定位于细胞核,在人胚肾细胞HEK-293中,Apoptin定位于细胞质中。体外转导试验表明,构建的重组杆状病毒BV-Apoptin能高效转导HepG2细胞,Apoptin的表达水平与杆状病毒的感染剂量成正相关,当感染复数越高,表达Apoptin蛋白的细胞数越多。
3.重组杆状病毒BV-Apoptin在体外、体内的抑瘤作用
将重组杆状病毒BV-Apoptin分别以不同感染复数转导HepG2细胞,通过DNA-Ladder和TUNEL检测细胞凋亡。结果显示,重组杆状病毒BV-Apoptin能够诱导转导的人肝癌细胞HepG2的产生凋亡,并且BV-Apoptin诱导肿瘤细胞凋亡的能力呈剂量依赖性。实验利用异体同源肿瘤细胞系移植的方法复制了C57BL/6小鼠荷H22肿瘤模型,研究构建的重组杆状病毒BV-Apoptin的体内抑瘤效果。结果显示,注射BV-Apoptin能够明显抑制小鼠肿瘤的生长,同时也提高荷瘤小鼠的存活率。以上结果提示,重组杆状病毒BV-Apoptin有望成为治疗肿瘤的一种新的有效手段。
4.重组杆状病毒BV-MICDYS的构建
从载体pcDNA3.1(+)中酶切CMV启动子的表达框,插入转移载体pFast-VSV-G中VSV-G的下游,构建转移载体pFast-G-CMV;然后从质粒pBSK-MICRO酶切全长的microdystrophin基因,分别克隆到pFast-G-CMV中,构建质粒pFast-G-MICDYS。转化DH_(10)Bac大肠杆菌,通过蓝白斑菌落及抗性筛选,获得重组穿梭载体Bacmid-MICDYS,提取其基因组并转染sf9昆虫细胞,获得重组杆状病毒BV-MICDYS。
5.重组杆状病毒BV-MICDYS转导C2C12细胞
将构建的重组杆状病毒BV-MICDYS以不同的感染复数(50、100、200MOI)转导C2C12细胞,转导后48h检测microdystrophin基因的表达。结果显示,重组杆状病毒BV-MICDYS能高效转导C2C12细胞,呈现明显的剂量依赖性。结果还显示,相同感染复数的Ad-MICDYS感染的C2C12细胞中microdystrophin蛋白表达数量低于相同感染复数BV-MICDYS转导的C2C12细胞中microdystrophin蛋白表达数量,说明了在C2C12细胞中,杆状病毒有着比腺病毒更高的靶基因表达水平的能力。
6.体内研究重组杆状病毒BV-MICDYS在mdx TA中的转导
将重组杆状病毒BV-MICDYS分别以不同的剂量(10~(10)pfu/mL,10~9pfu/mL,10~8pfu/mL)注射到mdx TA,2周和8周后做肌肉组织冰冻切片,HE检测肌肉病理的改善情况,免疫组化检测microdystrophin蛋白的表达,同时做原位检测等长拉伸诱导损伤后TA肌肉收缩力恢复情况。试验结果表明,在注射后2周,重组杆状病毒BV-MICDYS能介导microdystrophin蛋白在小鼠体内的肌肉细胞中高效表达,并且表达水平其呈现剂量依赖性,能更好的改善肌肉病理状况。但重组杆状病毒在注射后8周,未检测到其治疗效果,显示重组杆状病毒BV-MICDYS较适用于DMD的短期治疗。
The baculovirus Autographa californica multiple Nucleopolyhedrovirus (AcMNPV) has been traditionally used as an efficient vehicle to over-express recombinant proteins in insect cells. Its host specificity was originally thought to be restricted to cells derived from arthropods. Recently, however, accumulating evidence has revealed that baculovirus, carrying mammalian cell-active promoters, is capable of transferring and effective expressing foreign genes in a variety of mammalian cells. Thanks to its highly efficient gene delivery mechanism, increasing interest has been shown in baculovirus as a novel vector for gene therapy. In addition, other potential advantages, including easy manipulation, high recombinant viral titers, simple scale-up, a large DNA insertion capacity, lack of replication in mammalian cells, and lack of toxicity, make this vector highly promising for gene therapy.
Apoptin, a protein derived from the chicken anemia virus, can induce programmed cell death in a large panel of human transformed and malignant cells, but not in normal, diploid cells. The intrinsic tumor specificity of Apoptin makes it a promising new tool for cancer gene therapy.
Duchenne musclar dystrophy (DMD) is an X-linked recessive disease with characteristic of progressive degeneration and necrosis in skeletal muscle. There is no effective treatment for DMD at present, gene therapy is a promising therapeutical approaches for the treatment of DMD. Howeve, DMD gene therapy is challenged by the viral vectors for their small insert capacity, replication-deficient and require helper virus functions for propagation and raising biosafety concerns. These all impede their clinical application. Enhancement the experssed efficiency in vivo and selection ideal vector are the keys to success to treat DMD.
Based on the above mentioned research background, the present study investigate the anti-tumor effects in vitro and in vivo of a recombinant baculovirus encoding Apoptin protein, evaluated the microdystrophin protein expression of the recombinant baculovirus containing microdystrophin gene, at last, compared the gene therapy ability of the recombinant baculovirus and recombinant adenovirus in mdx mice. The main projects are:
1. Construction of recombinant baculovirus expressing the Apoptin protein
Full-length cDNA for Apoptin was amplified by polymerase chain reaction from plasmid pVAX-Apoptin, and subcloned into pcDNA-HisB vector to generate pcDNA3.1-His-Apoptin. The DNA fragment containing CMV-IE enhancer/promoter-Apoptin was obtained from pc-Apoptin and inserted into pFast-VSV-G to generate pFB-G-Apoptin. The recombinant baculoviruses were subsequently generated by using the Bac-to-Bac(?) system (Invitrogen) following the manufacturer's instructions. The virus was further amplified by propagation in Sf-9 cells.
2. The research on effect of transduction with recombinant baculovirus BV-Apoptin
We investigate the correct subcellular localization of BV-Apoptin in cells. We found that Apoptin was mainly located in the nucleus of HepG2 cells, whereas in normal HEK293 cells BV-Apoptin was localized in the cytoplasm. The results indicated that BV-Apoptin could mediate efficient gene delivery and the greater gene transduction efficiency of the BV-Apoptin was confirmed by comparing the Apoptin expression level in a variety of multiplity of infection.
3. The antitumor effect of BV-Apoptin in vitro and in vivo
To determine whether BV-Apoptin can induce apoptosis in transduced HepG2 cells, the DNA ladder and TUNEL assays were performed. An obvious DNA ladder pattern could be observed in BV-Apoptin-transduced HepG2 cells. The results indicated that BV-Apoptin induced apoptosis in a dose-dependent manner, specifically in transduced tumor cells but not in transduced normal cells. C57BL/6 mice model bearing H22 hepatoma was constructed by transplanting H22 cells into the right hind limb of the mice and the anti-tumor effect on H22 hepatoma of BV-Apoptin was observed. The results indicated that the treatment with BV-Apoptin can confer significant survival benefits and reduction of tumor size in vivo. Taken altogether, BV-Apoptin shows the good anti-tumor effect may be used as new and powerful strategy for cancer bio-therapy in future.
4. Construction of recombinant baculovirus expressing the microdystrophin protein
The DNA fragment containing CMV-IE enhancer/promoter was released from pcDNA3.1 (+) and inserted into pFast-VSV-G, resulting in pFB-G-CMV. Full-length cDNA for human microdystrophin gene was acqured from the plasmid pBSK-MICRO and subcloned into pFB-G-CMV to generate pFB-G-MICDYS. The recombinant baculoviruses were subsequently generated by using the Bac-to-Bac(?) system (Invitrogen) following the manufacturer's instructions. The virus was further amplified by propagation in Sf-9 cells.
5. Research on the effect of transduction with BV-MICDYS in C2C12 cells
The C2C12 cells were transduced with BV-Apoptin at different MOIs (50, 100 and 200). At 48 h after infection, the expression of microdystrophin was detected. Obvious differences in number of positive fluorescence cells were observed between wells infected with 50,100, 200 MOI of BV-MICDYS as the progression of the transduction. The microdystrophin protein expression by BV-MICDYS is in a dose-dependent manner in C2C12 cells. The results also indicated that in the same dosage, the microdystrophin positive of BV-MICDYS is higher than the microdystrophin expression with recombinant adenovirus Ad-MICDYS. These results indicated that BV-MICDYS could mediate efficient gene delivery and expression of microdystrophin protein in a dose-dependent manner in mammalian cells, and the transduction efficiency was higher than adenovirus.
6. Research on the effect of transduction with recombinant baculovirus mediated microdystrophin in mdx mice
TA muscles of mdx mice were injected with 10~(10)pfu/mL, 10~9pfu/mL, 10~8pfu/mL of recombinant baculovirus BV-MICDYS. Cryostat sections were prepared from the snap frozen muscle samples after 2 weeks and 8 weeks injection. HE evaluated pathological change of TA and immunocytochemistry evaluated the expression of microdystrophin; at the same time, the isometric contractile properties of TA were tested in situ. The results indicated in 2 weeks that transduction of the microdystrophin BV-MICDYS could retard significantly the degeneration and distribution in TA of mdx mice. The gene therapy ability of BV-MICDYS is in a dose-dependent pattern. But the BV-MICDYS lost its ability in gene therapy ability 8 weeks after injection. These results indicated that BV-MICDYS are suit for DMD short-term gene therapy.
引文
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