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

• मुख्य लेखकों: Patricia Peking (लेखक), Ulrich Koller (लेखक), Stefan Hainzl (लेखक), Sophie Kitzmueller (लेखक), Thomas Kocher (लेखक), Elisabeth Mayr (लेखक), Alexander Nyström (लेखक), Thomas Lener (लेखक), Julia Reichelt (लेखक), Johann W Bauer (लेखक), Eva M Murauer (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2016-01-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...