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

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

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...

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Bibliographic Details
Main Authors: Patricia Peking, Ulrich Koller, Stefan Hainzl, Sophie Kitzmueller, Thomas Kocher, Elisabeth Mayr, Alexander Nyström, Thomas Lener, Julia Reichelt, Johann W Bauer, Eva M Murauer
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
epidermolysis bullosa
in situ therapy
nonviral minicircle plasmid
RNA trans-splicing correction
type VII collagen
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117300811

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http://www.sciencedirect.com/science/article/pii/S2162253117300811

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