Gene correction therapy by homologous recombination

2014 Fiscal Year Final Research Report

• Project/Area Number: 24659265
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Applied pharmacology
• Research Institution: Nippon Medical School (2014); 独立行政法人国立精神・神経医療研究センター (2012-2013)
• Principal Investigator: OKADA Takashi 日本医科大学, 医学(系)研究科(研究院), 教授 (00326828)
• Project Period (FY): 2012-04-01 – 2015-03-31
• Keywords: 遺伝子修正 / 遺伝性神経筋疾患 / 細胞治療

Outline of Final Research Achievements

Duchenne muscular dystrophy (DMD) is the most common form of childhood muscular dystrophy. Although current gene therapy strategies aim at protein supplementation to recover the function, to correct the mutation in the dystrophin gene must be much-awaited technology as an ultimate goal. Also, since correction by gene editing requires the stable transfer of a functional gene into the target cell, we investigated mesenchymal stromal cells and dental pulp stem cells as platforms of gene and cell therapeutics for the treatment of DMD.

Free Research Field

脳神経外科学