A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice-Reference-Cited by-同舟云学术

A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice

Published:2013-03 Issue:3 Volume:24 Page:331-342
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Zhou Haiyan¹,Janghra Narinder¹,Mitrpant Chalermchai²³,Dickinson Rachel L.⁴,Anthony Karen¹,Price Loren³,Eperon Ian C.⁴,Wilton Stephen D.³,Morgan Jennifer¹,Muntoni Francesco¹

Affiliation:

1. Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, WC1N 1EH, United Kingdom.

2. Department of Biochemistry, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok 10700, Thailand.

3. Centre for Neuromuscular and Neurological Disorders (M518), University of Western Australia, Western Australia 6008, Australia.

4. Department of Biochemistry, University of Leicester, Leicester LE1 9HN, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2012.211

Reference62 articles.

1. Exonic Sequences Provide Better Targets for Antisense Oligonucleotides Than Splice Site Sequences in the Modulation of Duchenne Muscular Dystrophy Splicing

2. Efficacy of apolipoprotein B synthesis inhibition in subjects with mild-to-moderate hyperlipidaemia

3. Vascular Perfusion Abnormalities in Infants with Spinal Muscular Atrophy

4. Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy

5. Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy

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