scholarly journals Rescue of the Mucocutaneous Manifestations by Human Cord Blood Derived Nonhematopoietic Stem Cells in a Mouse Model of Recessive Dystrophic Epidermolysis Bullosa

Stem Cells ◽  
2015 ◽  
Vol 33 (6) ◽  
pp. 1807-1817 ◽  
Author(s):  
Yanling Liao ◽  
Larisa Ivanova ◽  
Hongwen Zhu ◽  
Ashlin Yahr ◽  
Janet Ayello ◽  
...  
Keyword(s):  
Stem Cells ◽  
Mouse Model ◽  
Cord Blood ◽  
Epidermolysis Bullosa ◽  
Human Cord Blood ◽  
Dystrophic Epidermolysis Bullosa ◽  
Recessive Dystrophic Epidermolysis Bullosa

scholarly journals Cord Blood-Derived Stem Cells Suppress Fibrosis and May Prevent Malignant Progression in Recessive Dystrophic Epidermolysis Bullosa

Stem Cells ◽  
2018 ◽  
Vol 36 (12) ◽  
pp. 1839-1850 ◽  
Author(s):  
Yanling Liao ◽  
Larisa Ivanova ◽  
Hongwen Zhu ◽  
Trevor Plumer ◽  
Carl Hamby ◽  
...  
Keyword(s):  
Stem Cells ◽  
Cord Blood ◽  
Epidermolysis Bullosa ◽  
Malignant Progression ◽  
Dystrophic Epidermolysis Bullosa ◽  
Recessive Dystrophic Epidermolysis Bullosa

scholarly journals Efficient in vivo gene editing using ribonucleoproteins in skin stem cells of recessive dystrophic epidermolysis bullosa mouse model

2017 ◽  
Vol 114 (7) ◽  
pp. 1660-1665 ◽  
Author(s):  
Wenbo Wu ◽  
Zhiwei Lu ◽  
Fei Li ◽  
Wenjie Wang ◽  
Nannan Qian ◽  
...  
Keyword(s):  
Stem Cells ◽  
Mouse Model ◽  
Genome Editing ◽  
Epidermolysis Bullosa ◽  
Mammalian Cells ◽  
Gene Editing ◽  
Genetic Diseases ◽  
Dystrophic Epidermolysis Bullosa ◽  
Recessive Dystrophic Epidermolysis Bullosa

The prokaryotic CRISPR/Cas9 system has recently emerged as a powerful tool for genome editing in mammalian cells with the potential to bring curative therapies to patients with genetic diseases. However, efficient in vivo delivery of this genome editing machinery and indeed the very feasibility of using these techniques in vivo remain challenging for most tissue types. Here, we show that nonreplicable Cas9/sgRNA ribonucleoproteins can be used to correct genetic defects in skin stem cells of postnatal recessive dystrophic epidermolysis bullosa (RDEB) mice. We developed a method to locally deliver Cas9/sgRNA ribonucleoproteins into the skin of postnatal mice. This method results in rapid gene editing in epidermal stem cells. Using this method, we show that Cas9/sgRNA ribonucleoproteins efficiently excise exon80, which covers the point mutation in our RDEB mouse model, and thus restores the correct localization of the collagen VII protein in vivo. The skin blistering phenotype is also significantly ameliorated after treatment. This study provides an in vivo gene correction strategy using ribonucleoproteins as curative treatment for genetic diseases in skin and potentially in other somatic tissues.

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