mRNAtrans -splicing in gene therapy for genetic diseases

Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3′ splice sites

(2015) Susanne Philippi et al.   HUMAN MOLECULAR GENETICS

Repair of Rhodopsin mRNA by Spliceosome-Mediated RNA Trans -Splicing: A New Approach for Autosomal Dominant Retinitis Pigmentosa

(2015) Adeline Berger et al.   MOLECULAR THERAPY

Gene therapy: progress and predictions

(2015) Mary Collins et al.   PROCEEDINGS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES

Trans-splicing repair of mutant p53 suppresses the growth of hepatocellular carcinoma cells in vitro and in vivo

(2015) Xingxing He et al.   Scientific Reports

Application of CRISPR/Cas9 for biomedical discoveries

(2015) Sean M. Riordan et al.   Cell and Bioscience

Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies

(2015) Lisa M. Ott de Bruin et al.   Frontiers in Immunology

Functional repair of p53 mutation in colorectal cancer cells using trans-splicing

(2015) Xingxing He et al.   Oncotarget

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

A day in the life of the spliceosome

(2014) A. Gregory Matera et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

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

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

Integrative transcriptome sequencing identifies trans-splicing events with important roles in human embryonic stem cell pluripotency

(2013) C.-S. Wu et al.   GENOME RESEARCH

Trans-splicing correction of tau isoform imbalance in a mouse model of tau mis-splicing

(2013) María Elena Avale et al.   HUMAN MOLECULAR GENETICS

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

Replacement of huntingtin exon 1 by trans-splicing

(2012) Hansjörg Rindt et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

A complex network of factors with overlapping affinities represses splicing through intronic elements

(2012) Yang Wang et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Intronic splicing enhancers, cognate splicing factors and context-dependent regulation rules

(2012) Yang Wang et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Exon and intron definition in pre-mRNA splicing

(2012) Laura De Conti et al.   Wiley Interdisciplinary Reviews-RNA

Optimization of SMN Trans-Splicing Through the Analysis of SMN Introns

(2011) Monir Shababi et al.   JOURNAL OF MOLECULAR NEUROSCIENCE

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

Exon Exchange Approach to Repair Duchenne Dystrophin Transcripts

(2010) Stéphanie Lorain et al.   PLoS One

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

(2008) H Y Chen et al.   GENE THERAPY

Trans-splicing Into Highly Abundant Albumin Transcripts for Production of Therapeutic Proteins In Vivo

(2008) Jun Wang et al.   MOLECULAR THERAPY

Artificial MicroRNAs as siRNA Shuttles: Improved Safety as Compared to shRNAs In vitro and In vivo

(2008) Ryan L Boudreau et al.   MOLECULAR THERAPY

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

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

5′ Trans-Splicing Repair of the PLEC1 Gene

(2007) Verena Wally et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY