Correction of the Middle Eastern M712T Mutation Causing GNE Myopathy by Trans-Splicing

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

Replacement of huntingtin exon 1 by trans-splicing

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

Sustained expression and safety of human GNE in normal mice after gene transfer based on AAV8 systemic delivery

(2012) Stella Mitrani-Rosenbaum et al.   NEUROMUSCULAR DISORDERS

The Proteomic Profile of Hereditary Inclusion Body Myopathy

(2011) Ilan Sela et al.   PLoS One

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

Hereditary Inclusion Body Myopathy: A decade of progress

(2009) Marjan Huizing et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE