Watson Deborah J, Passini Marco A, Wolfe John H
Department of Pathobiology and Walter Flato Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Hum Gene Ther. 2005 Jan;16(1):49-56. doi: 10.1089/hum.2005.16.49.
Evaluation of gene transfer into the developing mouse brain has shown that when adeno-associated virus serotype 1 (AAV1) or AAV2 vectors are injected into the cerebral lateral ventricles at birth, widespread parenchymal transduction occurs. Lentiviral vectors have not been tested by this route. In this study, we found that injection of lentiviral vectors pseudotyped with vesicular stomatitis virus glycoprotein (VSV-G) resulted in targeted transduction of the ependymal cells lining the ventricular system and the choroid plexus along the entire rostrocaudal axis of the brain, whereas a Mokola pseudotype transduced only a few cells after injection into the neonatal ventricle. In contrast, when lentiviral vectors pseudotyped with either VSV-G or Mokola glycoprotein are injected into the adult mouse brain, they transduce similar patterns of cells. An Ebola-Zaire-pseudotyped vector did not transduce any neonatal CNS cells, as was also the case for adult parenchymal injections. Long-term gene expression (12 months) occurred with a constitutively active mammalian promoter and a self-inactivating long terminal repeat (LTR), whereas the cytomegalovirus promoter in a vector with an intact LTR was expressed only in short-term experiments. We found that an AAV5 vector also targeted the ependymal and choroid plexus cells throughout the ventricular system. This vector exhibited limited penetration from the ventricle to other structures, which was significantly different from the previously reported patterns of transduction after intraventricular injection of AAV1 and AAV2 vectors.
对基因导入发育中小鼠大脑的评估表明,当在出生时将1型腺相关病毒(AAV1)或AAV2载体注入大脑侧脑室时,会发生广泛的实质转导。慢病毒载体尚未通过此途径进行测试。在本研究中,我们发现注射水疱性口炎病毒糖蛋白(VSV-G)假型化的慢病毒载体可导致沿脑室系统内衬的室管膜细胞和脉络丛在大脑整个头尾轴上的靶向转导,而莫科拉假型在注入新生脑室后仅转导少数细胞。相比之下,当将VSV-G或莫科拉糖蛋白假型化的慢病毒载体注入成年小鼠大脑时,它们转导的细胞模式相似。埃博拉-扎伊尔假型载体未转导任何新生中枢神经系统细胞,成年实质注射也是如此。使用组成型活性哺乳动物启动子和自失活长末端重复序列(LTR)可实现长期基因表达(12个月),而在具有完整LTR的载体中,巨细胞病毒启动子仅在短期实验中表达。我们发现AAV5载体也靶向整个脑室系统的室管膜和脉络丛细胞。该载体从脑室向其他结构的穿透有限,这与先前报道的脑室内注射AAV1和AAV2载体后的转导模式有显著差异。