An exon-specific U1 small nuclear RNA (snRNA) strategy to correct splicing defects

Gene Therapeutic Approach Using Mutation-adapted U1 snRNA to Correct a RPGR Splice Defect in Patient-derived Cells

(2011) Esther Glaus et al.   MOLECULAR THERAPY

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

(2010) Y. Hua et al.   GENES & DEVELOPMENT

Spinal muscular atrophy: mechanisms and therapeutic strategies

(2010) C. L. Lorson et al.   HUMAN MOLECULAR GENETICS

Excessive RNA Splicing and Inhibition of HIV-1 Replication Induced by Modified U1 Small Nuclear RNAs

(2010) D. Mandal et al.   JOURNAL OF VIROLOGY

Expansion of the eukaryotic proteome by alternative splicing

(2010) Timothy W. Nilsen et al.   NATURE

Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs

(2010) Virginie François et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

The transcription factor E2F1 and the SR protein SC35 control the ratio of pro-angiogenic versus antiangiogenic isoforms of vascular endothelial growth factor-A to inhibit neovascularization in vivo

(2010) G Merdzhanova et al.   ONCOGENE

Pro-metastatic splicing of Ron proto-oncogene mRNA can be reversed: Therapeutic potential of bifunctional oligonucleotides and indole derivatives

(2010) Claudia Ghigna et al.   RNA Biology

Rescue of coagulation factor VII function by the U1+5A snRNA

(2009) M. Pinotti et al.   BLOOD

Activating the synthesis of progerin, the mutant prelamin A in Hutchinson–Gilford progeria syndrome, with antisense oligonucleotides

(2009) Loren G. Fong et al.   HUMAN MOLECULAR GENETICS

Major differences in bleeding symptoms between factor VII deficiency and hemophilia B

(2009) F. BERNARDI et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Recognition of atypical 5′ splice sites by shifted base-pairing to U1 snRNA

(2009) Xavier Roca et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice

(2008) Yimin Hua et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation

(2008) Kathrin Meyer et al.   HUMAN MOLECULAR GENETICS

Therapeutic strategy to rescue mutation-induced exon skipping in rhodopsin by adaptation of U1 snRNA

(2008) Gaby Tanner et al.   HUMAN MUTATION