Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice

被引：47

作者：

Fujita, Yasuyuki ^{[1
]}

Abe, Riichiro ^{[1
]}

Inokuma, Daisuke ^{[1
]}

Sasaki, Mikako ^{[1
]}

Hoshina, Daichi ^{[1
]}

Natsuga, Ken ^{[1
]}

Nishie, Wataru ^{[1
]}

McMillan, James R. ^{[1
]}

Nakamura, Hideki ^{[1
]}

Shimizu, Tadamichi ^{[2
]}

Akiyama, Masashi ^{[1
]}

Sawamura, Daisuke ^{[3
]}

Shimizu, Hiroshi ^{[1
]}

机构：

[1] Hokkaido Univ, Grad Sch Med, Dept Dermatol, Sapporo, Hokkaido 0608638, Japan

[2] Toyama Univ, Grad Sch Med & Pharmaceut Sci, Dept Dermatol, Toyama 9300194, Japan

[3] Hirosaki Univ, Grad Sch Med, Dept Dermatol, Hirosaki, Aomori 0368562, Japan

来源：

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA | 2010年 / 107卷 / 32期

关键词：

hematopoietic stem cells; type XVII collagen; MESENCHYMAL STEM-CELLS; VII COLLAGEN; MOUSE MODEL; SKIN; GENE; DISEASE; REPAIR; REGENERATION; FIBROBLASTS; REPLACEMENT;

D O I：

10.1073/pnas.1000044107

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

Attempts to treat congenital protein deficiencies using bone marrow-derived cells have been reported. These efforts have been based on the concepts of stem cell plasticity. However, it is considered more difficult to restore structural proteins than to restore secretory enzymes. This study aims to clarify whether bone marrow transplantation (BMT) treatment can rescue epidermolysis bullosa (EB) caused by defects in keratinocyte structural proteins. BMT treatment of adult collagen XVII (Col17) knockout mice induced donor-derived keratinocytes and Col17 expression associated with the recovery of hemidesmosomal structure and better skin manifestations, as well improving the survival rate. Both hematopoietic and mesenchymal stem cells have the potential to produce Col17 in the BMT treatment model. Furthermore, human cord blood CD34(+) cells also differentiated into keratinocytes and expressed human skin component proteins in transplanted immunocompromised (NOD/SCID/gamma(null)(c)) mice. The current conventional BMT techniques have significant potential as a systemic therapeutic approach for the treatment of human EB.

引用

页码：14345 / 14350

页数：6

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