Article
CRISPR/Cas9-Mediated In Situ Correction of LAMB3 Gene in Keratinocytes Derived from a Junctional Epidermolysis Bullosa Patient.
Molecular therapy : the journal of the American Society of Gene Therapy - 7 Nov 2018
Benati Daniela, Miselli Francesca, Cocchiarella Fabienne, Patrizi Clarissa, Carretero Marta, Baldassarri Samantha, Ammendola Virginia, Has Cristina, Colloca Stefano, Del Rio Marcela, Larcher Fernando, Recchia Alessandra
Abstract excerpt
Deficiency of basement membrane heterotrimeric laminin 332 component, coded by LAMA3, LAMB3, and LAMC2 genes, causes junctional epidermolysis bullosa (JEB), a severe skin adhesion defect. Herein, we report the first application of CRISPR/Cas9-mediated homology direct repair (HDR) to in situ restore LAMB3 expression in JEB keratinocytes in vitro and in immunodeficient mice transplanted with genetically corrected...
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