Construction and validation of an RNA trans-splicing molecule suitable to repair a large number of COL7A1 mutations

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

(2016) Patricia Peking et al.   Molecular Therapy-Nucleic Acids

Trans-Splicing Improvement by the Combined Application of Antisense Strategies

(2015) Ulrich Koller et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Potential of Systemic Allogeneic Mesenchymal Stromal Cell Therapy for Children with Recessive Dystrophic Epidermolysis Bullosa

(2015) Gabriela Petrof et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Inherited epidermolysis bullosa: Updated recommendations on diagnosis and classification

(2014) Jo-David Fine et al.   JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY

Cis -splicing and Translation of the Pre- Trans -splicing Molecule Combine With Efficiency in Spliceosome-mediated RNA Trans -splicing

(2014) François Monjaret et al.   MOLECULAR THERAPY

mRNA-based therapeutics — developing a new class of drugs

(2014) Ugur Sahin et al.   NATURE REVIEWS DRUG DISCOVERY

Considerations for a Successful RNA Trans-splicing Repair of Genetic Disorders

(2014) Ulrich Koller et al.   Molecular Therapy-Nucleic Acids

Intravenously Injected Recombinant Human Type VII Collagen Homes to Skin Wounds and Restores Skin Integrity of Dystrophic Epidermolysis Bullosa

(2013) David T. Woodley et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Dystrophin rescue by trans -splicing: a strategy for DMD genotypes not eligible for exon skipping approaches

(2013) Stéphanie Lorain et al.   NUCLEIC ACIDS RESEARCH

A Reporter-Based Screen to Identify Potent 3’ Trans-Splicing Molecules for Endogenous RNA Repair

(2013) Eva M. Murauer et al.   Human Gene Therapy Methods

Promoter Targeting shRNA Suppresses HIV-1 Infection In vivo Through Transcriptional Gene Silencing

(2013) Kazuo Suzuki et al.   Molecular Therapy-Nucleic Acids

Microneedle delivery of plasmid DNA to living human skin: Formulation coating, skin insertion and gene expression

(2012) Marc Pearton et al.   JOURNAL OF CONTROLLED RELEASE

Spliceosome-Mediated Trans-Splicing: The Therapeutic Cut and Paste

(2012) Verena Wally et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

TheCOL7A1mutation database

(2011) Katarzyna Wertheim-Tysarowska et al.   HUMAN MUTATION

A novel screening system improves genetic correction by internal exon replacement

(2011) U. Koller et al.   NUCLEIC ACIDS RESEARCH

K14 mRNA reprogramming for dominant epidermolysis bullosa simplex

(2010) Verena Wally et al.   HUMAN MOLECULAR GENETICS

Functional Correction of Type VII Collagen Expression in Dystrophic Epidermolysis Bullosa

(2010) Eva M. Murauer et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Trans-Splicing-Mediated Improvement in a Severe Mouse Model of Spinal Muscular Atrophy

(2010) T. H. Coady et al.   JOURNAL OF NEUROSCIENCE

SIN Retroviral Vectors Expressing COL7A1 Under Human Promoters for Ex Vivo Gene Therapy of Recessive Dystrophic Epidermolysis Bullosa

(2010) Matthias Titeux et al.   MOLECULAR THERAPY

A Transposon and Transposase System for Human Application

(2010) Perry B Hackett et al.   MOLECULAR THERAPY

Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa

(2010) John E. Wagner et al.   NEW ENGLAND JOURNAL OF MEDICINE

Correction of tau mis-splicing caused by FTDP-17 MAPT mutations by spliceosome-mediated RNA trans-splicing

(2009) T. Rodriguez-Martin et al.   HUMAN MOLECULAR GENETICS

Correction of dystrophia myotonica type 1 pre-mRNA transcripts by artificial trans-splicing

(2008) H Y Chen et al.   GENE THERAPY

Potential of Fibroblast Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa

(2008) Tracy Wong et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Development of a Single Vector System that Enhances Trans-Splicing of SMN2 Transcripts

(2008) Tristan H. Coady et al.   PLoS One