Sci Transl Mediterranean 2014 Umegaki Arao 264ra164

 Sci Transl Med 2014 Umegaki Arao 264ra164 Exploration Paper

Activated pluripotent stem cells from human revertant keratinocytes for the treatment of epidermolysis bullosa

Noriko Umegaki-Arao ain al.

Sci Transl Scientif 6, 264ra164 (2014);

DOI: 10. 1126/scitranslmed. 3009342

Editor's Summary

Epidermolysis bullosa (EB) is a unusual, inherited skin disorder that creates such serious blistering that patients in many cases are relegated into a delicate life in bandages. Like a miscuglio quilt, your skin of a patient with EB can consist of both mutated skin cells (which trigger the disease) and spontaneously genetically remedied ''normal'' cells; this patchwork phenomenon is recognized as revertant mosaicism. In a new study, Umegaki-Arao and colleagues demonstrated that these revertant cells could be used to generate healthful skin, addressing a possible cell therapy to get patients with EB who may have no treatments. The authors took revertant keratinocytes (skin cells) by a patient with junctional EB, who have changement in the gene expressing type XVII collagen. These revertant keratinocytes had been used to create induced pluripotent stem skin cells, which, consequently, could be differentiated into a keratinocyte lineage that created normal-looking skin layers not only in vitro but as well in feston in mice. Because the cells already expressed type XVII collagen, there were no need for genetic correction, therefore avoiding lots of the pitfalls that gene and cell therapies face during translation to the clinic.

An entire electronic variation of this article and also other services, which include high-resolution statistics, can be found at:

http://stm.sciencemag.org/content/6/264/264ra164.full.html

Supplementary Material can be found in the web version of this article at: http://stm.sciencemag.org/content/suppl/2014/11/24/6.264.264ra164.DC1.html Related Resources for this article are available online at:

http://stm.sciencemag.org/content/scitransmed/6/264/264ra163.full.html http://stm.sciencemag.org/content/scitransmed/6/264/264ra165.full.html http://stm.sciencemag.org/content/scitransmed/5/178/178fs10.full.html http://stm.sciencemag.org/content/scitransmed/4/149/149fs31.full.html http://stm.sciencemag.org/content/scitransmed/4/127/127ps9.full.html http://stm.sciencemag.org/content/scitransmed/5/179/179ps7.full.html Information about obtaining reprints of this article or regarding obtaining permission to recreate this article in whole or simply can be found at:

http://www.sciencemag.org/about/permissions.dtl

Science Translational Medicine (print ISSN 1946-6234; online ISSN 1946-6242) can be published regular, except the very last week in December, by American Connection for the Advancement of Science, 1200 New York Method NW, Washington, DC 20005. Copyright 2014 by the American Association intended for the Growth of Scientific research; all rights reserved. The title Science Translational Medicine can be described as registered brand of AAAS.

Downloaded via stm. sciencemag. org about December one particular, 2014

''Natural Gene Therapy'' for Rare, Genetic Skin condition

RESEARCH CONTENT

SKIN DISEASE

Caused pluripotent control cells from human

revertant keratinocytes pertaining to the treatment of

epidermolysis bullosa

Revertant mosaicism is known as a naturally occurring phenomenon involving natural correction of a pathogenic gene mutation within a somatic cellular. It has been noticed in several innate diseases, including epidermolysis bullosa (EB), a team of inherited skin disorders characterized by blistering and scarring damage. Induced pluripotent stem cellular material (iPSCs), made from fibroblasts or keratinocytes, have been proposed as a treatment for EB. However , this requires genome editing and enhancing to correct the mutations, and, in gene therapy, productivity of targeted gene correction and bad genomic changes are still limits of translation. We display the technology of iPSCs from revertant keratinocytes of a junctional EB patient with compound heterozygous COL17A1 changement. These revertant iPSCs were then differentiated into naturally genetically remedied keratinocytes...

References: Mechanisms of natural gene therapy. Discov. Med. 13, 167–179 (2012).

161, 444–447 (2009).

several. M. N. Jonkman, Revertant mosaicism in human genetic disorders. Am. J. Scientif. Genet. 85,

361–364 (1999).

Genet. 16, 293–303 (1988).

Dermatol. 75, 1103–1126 (2014).

Ther. 21 years old, 1299–1310 (2010).

J. Tolar, TALEN-based gene correction for epidermolysis bullosa. Mol. Ther. 21, 1151–1159 (2013).

dystrophic epidermolysis bullosa. J. Make investments. Dermatol. 128, 2179–2189 (2008).

J. Spend. Dermatol. 118, 626–630 (2002).

Invest. 117, 1240–1248 (2007).

Cell 88, 543–551 (1997).

Hum. Genet. 77, 727–740 (2005).

bullosa. J. Make investments. Dermatol. 130, 1937–1940 (2010).

Med. fish hunter 360, 1680–1682 (2009).

mutations. M. Invest. Dermatol. 132, 1374–1383 (2012).

twenty three. L. To. Cheng, L. T. Sunlight, T. Tada, Genome croping and editing in activated pluripotent come cells. Family genes

Cells seventeen, 431–438 (2012).

24. A. Gostyński, A. M. Pasmooij, M. N. Jonkman, Powerful therapeutic hair transplant of revertant skin in epidermolysis bullosa. J. I am. Acad. Dermatol. 70, 98–101 (2014).

collagen revertant cellular material in an creature model of revertant cell remedy. J. Invest. Dermatol. 134,

571–574 (2014).

Acad. Sci. U. S i9000. A. 108, 8797–8802 (2011).

1145–1147 (1998).

6, 264ra165 (2014).

Downloaded from stm. sciencemag. org on December 1, 2014

RESEARCH ARTICLE

PLOS One 8, e77673 (2013).


Related

Evaluating Biometric System Article

An intro to Assessing Biometric Systems How and where biometric systems are deployed would depend on their overall performance. Knowing what might and how to decipher the answers…...

YOSHIMOTO CLOWN Research Paper

YOSHIMOTO BANANA An Introduction The first book I actually ever go through and fell in love with was a novel written by Virgina Andrews called ‘Dark Angel'.…...

ADHD and Auditory procedure Essay

Launch Nurses, related health professionals, educational institutions and the public have observed a dramatic increase in Oral Process Disorder (APD) and Attention-Deficit Over activity disorder (ADHD) in school-age…...

Labeling Composition Sociology

Each time a person can be labeled as something he or she has become part of that particular sub-culture, and although the face might not consider themselves to be that…...

REACTION PAPER ON ARCTIC

In comparison with the native people in the Arctic for thousands of years, European explorers are family member tenderfoots. That they came to the Arctic for colonization. That they started…...

Essay on Body Image in Advertising, a Two Faced Ind

Erica K. Culler Professor Mandsperson Renchen English language 100 Section 84 of sixteen October 2012 Body Image in Advertising, a Two-Faced Industry Body…...