Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes

• Main Authors: Cristina Chamorro (Author), Angeles Mencía (Author), David Almarza (Author), Blanca Duarte (Author), Hildegard Büning (Author), Jessica Sallach (Author), Ingrid Hausser (Author), Marcela Del Río (Author), Fernando Larcher (Author), Rodolfo Murillas (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Clonal gene therapy protocols based on the precise manipulation of epidermal stem cells require highly efficient gene-editing molecular tools. We have combined adeno-associated virus (AAV)-mediated delivery of donor template DNA with transcription activator-like nucleases (TALE) expressed by adenovi...