title: RNA trans-splicing for genodermatoses.
abstract: Spliceosome-mediated RNA trans-splicing (SMaRT) is a tool that facilitates the recombination of two distinct pre-mRNA molecules. Its application for gene therapeutic purposes has been hindered by laborious procedures to identify gene-specific molecules. We have established a screening method for the identification of highly functional RNA trans-splicing molecules based on fluorescence reporters, facilitating the generation of most potent therapeutic molecules for the correction of any gene of interest.
authors: Division of Molecular Dermatology and EB House Austria, Department of Dermatology, Paracelsus Private Medical University, Salzburg, Austria. jo.bauer@salk.at
authors: Johann W
authors: JW
authors: Bauer
authors: Eva M
authors: EM
authors: Murauer
authors: Verena
authors: V
authors: Wally
authors: Ulrich
authors: U
authors: Koller
mesh_terms: D000818:Animals; D001483:Base Sequence; D018929:Cell Culture Techniques / Q000379:methods; D002460:Cell Line; D003001:Cloning, Molecular / Q000379:methods; D017931:DNA Primers / Q000235:genetics; D005434:Flow Cytometry / Q000379:methods; D017930:Genes, Reporter; D015316:Genetic Therapy / Q000379:methods*; D049452:Green Fluorescent Proteins / Q000235:genetics; D006801:Humans; D008164:Luminescent Proteins / Q000235:genetics; D008969:Molecular Sequence Data; D016133:Polymerase Chain Reaction / Q000379:methods; D012333:RNA, Messenger / Q000235:genetics; D012867:Skin / Q000378:metabolism; D012871:Skin Diseases / Q000235:genetics* / Q000628:therapy; D017381:Spliceosomes / Q000235:genetics; D020040:Trans-Splicing; D014162:Transfection
chemical_list: D017931:DNA Primers; D008164:Luminescent Proteins; D012333:RNA, Messenger; C413663:fluorescent protein 583; D049452:Green Fluorescent Proteins