2010 Fiscal Year Final Research Report

Lentiviral vector mediated gene therapy for Krabbe disease

Research Project

Project/Area Number	20591233
Research Category	Grant-in-Aid for Scientific Research (C)
Allocation Type	Single-year Grants
Section	一般
Research Field	Pediatrics
Research Institution	Jikei University School of Medicine
Principal Investigator	KOBAYASHI Hiroshi Jikei University School of Medicine, 医学部, 講師 (90266619)
Co-Investigator(Kenkyū-buntansha)	KIMURA Takahiro 東京慈恵会医科大学, 医学部, 助教 (00307430) KAWAI Toshinao 東京慈恵会医科大学, 医学部, 助教 (20328305)
Project Period (FY)	2008 – 2010
Keywords	遺伝 / 先天異常学 / 遺伝子治療
Research Abstract	We investigated the effect of lenti-virus mediated gene therapy for Krabbe disease. We succeeded in preparing high titer lenti-virus including GALC, deficient enzyme in Krabbe disease expressing gene by large scale harvesting and ultracentrifuge, and injected for neonatal twitcher mouse, model for Krabbe disease resulted in efficient improvement in nutrition and life span. And we had more efficient improvement in the phenotype of twitcher mice by the combination of gene therapy and substrate reduction therapy using L-cycloserine.

(2 results)