Inhibition of Fibrillin 1 Expression Using U1 snRNA as a Vehicle for the Presentation of Antisense Targeting Sequence

By Montgomery Robert A.   Dietz Harry C.  

Human Molecular Genetics, Vol. 6, Iss. 4, 1997-01 ,pp. : 519-525

Oxford University Press

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

By Meyer Kathrin   Marquis Julien   Trb Judith   Nlend Nlend Rachel   Verp Sonia   Ruepp Marc-David   Imboden Hans   Barde Isabelle   Trono Didier   Schmperli Daniel  

Human Molecular Genetics, Vol. 18, Iss. 3, 2009-02 ,pp. : 546-555

Oxford University Press

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Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

By Goyenvalle Aurélie   Babbs Arran   Wright Jordan   Wilkins Vivienne   Powell Dave   Garcia Luis   Davies Kay E.  

Human Molecular Genetics, Vol. 21, Iss. 11, 2012-06 ,pp. : 2559-2571

Oxford University Press

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Mutations affecting mRNA splicing are the most common molecular defects in patients with neurofibromatosis type 1

By Ars Elisabet   Serra Eduard   García Judit   Kruyer Helena   Gaona Antonia   Lázaro Conxi   Estivill Xavier  

Human Molecular Genetics, Vol. 9, Iss. 2, 2000-01 ,pp. : 237-247

Oxford University Press

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Myotonic dystrophy type 1 is associated with nuclear foci of mutant RNA, sequestration of muscleblind proteins and deregulated alternative splicing in neurons

By Jiang Hong  

Human Molecular Genetics, Vol. 13, Iss. 24, 2004-12 ,pp. : 3079-3088

Oxford University Press

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