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2014 Fiscal Year Final Research Report

Gene Therapy for the Krabbe disease using zinc finger nuclease system

Research Project

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Project/Area Number 24591527
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Pediatrics
Research InstitutionJikei University School of Medicine

Principal Investigator

KOBAYASHI Hiroshi  東京慈恵会医科大学, 医学部, 准教授 (90266619)

Co-Investigator(Kenkyū-buntansha) YOTA Shimada  東京慈恵会医科大学, 総合医科学研究センター遺伝子治療研究部, 助教 (20560824)
Project Period (FY) 2012-04-01 – 2015-03-31
KeywordsKrabbe病 / Zinc Finger / GALC
Outline of Final Research Achievements

We prepared the specific donor plasmid (DP) and zinc finger nuclease (ZFN) for homologus recombination of the point mutation (chromosome 12, exon 10) in the model mouse of Krabbe disease, and co-transfected them into the Schwann cell line origin from the model mouse by the nucleofecta (electropolation system), resulted in normalize of the point mutation, efficient high expression of GALC enzyme (deficient in the Krabbe disease) activity, reduction of pscichosine in the treated cell, and it has been maintained after some passages.

Free Research Field

先天代謝異常

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Published: 2016-06-03  

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