A Gene Gun-mediated Nonviral RNA trans-splicing Strategy for Col7a1 Repair

• Main Authors: Patricia Peking (Author), Ulrich Koller (Author), Stefan Hainzl (Author), Sophie Kitzmueller (Author), Thomas Kocher (Author), Elisabeth Mayr (Author), Alexander Nyström (Author), Thomas Lener (Author), Julia Reichelt (Author), Johann W Bauer (Author), Eva M Murauer (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

RNA trans-splicing represents an auspicious option for the correction of genetic mutations at RNA level. Mutations within COL7A1 causing strong reduction or absence of type VII collagen are associated with the severe skin blistering disease dystrophic epidermolysis bullosa. The human COL7A1 mRNA con...