PMID- 20947660 OWN - NLM STAT- MEDLINE DCOM- 20111207 LR - 20240503 IS - 1460-2083 (Electronic) IS - 0964-6906 (Print) IS - 0964-6906 (Linking) VI - 20 IP - 2 DP - 2011 Jan 15 TI - Dystrophin conferral using human endothelium expressing HLA-E in the non-immunosuppressive murine model of Duchenne muscular dystrophy. PG - 235-44 LID - 10.1093/hmg/ddq458 [doi] AB - Human leukocyte antigen (HLA)-E is a non-classical major histocompatibility complex class I (Ib) molecule, which plays an important role in immunosuppression. In this study, we investigated the immunomodulating effect of HLA-E in a xenogeneic system, using human placental artery-derived endothelial (hPAE) cells expressing HLA-E in a mouse model. In vitro cell lysis analysis by primed lymphocytes in combination with siRNA transfection showed that HLA-E is necessary for inhibition of the immune response. Similarly, in vivo cell implantation analysis with siRNA-mediated down-regulation of HLA-E demonstrates that HLA-E is involved in immunosuppression. As hPAE cells efficiently transdifferentiate into myoblasts/myocytes in vitro, we transplanted the cells into mdx mice, a model of Duchenne muscular dystrophy. hPAE cells conferred dystrophin to myocytes of the 'immunocompetent' mdx mice with extremely high efficiency. These findings suggest that HLA-E-expressing cells with a myogenic potential represent a promising source for cell-based therapy of patients with muscular dystrophy. FAU - Cui, Chang-Hao AU - Cui CH AD - Department of Reproductive Biology, National Institute for Child Health and Development, Tokyo, Japan. FAU - Miyoshi, Shunichiro AU - Miyoshi S FAU - Tsuji, Hiroko AU - Tsuji H FAU - Makino, Hatsune AU - Makino H FAU - Kanzaki, Seiichi AU - Kanzaki S FAU - Kami, Daisuke AU - Kami D FAU - Terai, Masanori AU - Terai M FAU - Suzuki, Harumi AU - Suzuki H FAU - Umezawa, Akihiro AU - Umezawa A LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20101014 PL - England TA - Hum Mol Genet JT - Human molecular genetics JID - 9208958 RN - 0 (Dystrophin) RN - 0 (Histocompatibility Antigens Class I) RN - 0 (RNA, Small Interfering) SB - IM MH - Animals MH - Arteries/cytology MH - Cell Culture Techniques MH - Disease Models, Animal MH - Dystrophin/*genetics/*metabolism MH - *Endothelial Cells/metabolism MH - Gene Expression Regulation/immunology MH - Histocompatibility Antigens Class I/*genetics/metabolism MH - Humans MH - Immunocompetence/immunology MH - Mice MH - Mice, Inbred BALB C MH - Mice, Inbred mdx MH - Muscle Development/genetics MH - Muscular Dystrophy, Duchenne/*genetics/immunology/therapy MH - RNA, Small Interfering/genetics/metabolism MH - HLA-E Antigens PMC - PMC3005899 EDAT- 2010/10/16 06:00 MHDA- 2011/12/13 00:00 PMCR- 2010/10/14 CRDT- 2010/10/16 06:00 PHST- 2010/10/16 06:00 [entrez] PHST- 2010/10/16 06:00 [pubmed] PHST- 2011/12/13 00:00 [medline] PHST- 2010/10/14 00:00 [pmc-release] AID - ddq458 [pii] AID - 10.1093/hmg/ddq458 [doi] PST - ppublish SO - Hum Mol Genet. 2011 Jan 15;20(2):235-44. doi: 10.1093/hmg/ddq458. Epub 2010 Oct 14.