New developments in exon skipping and splice modulation therapies for neuromuscular diseases

A Touznik, JJA Lee, T Yokota - Expert opinion on biological …, 2014 - Taylor & Francis
Introduction: Antisense oligonucleotide (AON) therapy is a form of treatment for genetic or
infectious diseases using small, synthetic DNA-like molecules called AONs. Recent …
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[PDF][PDF] New developments in exon skipping and splice modulation therapies for neuromuscular diseases

A Touznik, JJA Lee, T Yokota - Expert opinion on biological …, 2014 - drive.google.com
Expert opinion: The main approach of AON therapy in DMD and SMA is to rescue ('knock
up'or increase) target proteins through exon skipping or exon inclusion; conversely, most …

[引用][C] New developments in exon skipping and splice modulation therapies for neuromuscular diseases

A Touznik, JJA Lee, T Yokota - Expert Opinion on Biological Therapy, 2014 - cir.nii.ac.jp
New developments in exon skipping and splice modulation therapies for neuromuscular
diseases | CiNii Research CiNii 国立情報学研究所 学術情報ナビゲータ[サイニィ] 詳細へ移動 …

New developments in exon skipping and splice modulation therapies for neuromuscular diseases

A Touznik, JJA Lee, T Yokota - Expert opinion on …, 2014 - pubmed.ncbi.nlm.nih.gov
Introduction Antisense oligonucleotide (AON) therapy is a form of treatment for genetic or
infectious diseases using small, synthetic DNA-like molecules called AONs. Recent …

New developments in exon skipping and splice modulation therapies for neuromuscular diseases.

A Touznik, JJ Lee, T Yokota - Expert Opinion on Biological Therapy, 2014 - europepmc.org
Antisense oligonucleotide (AON) therapy is a form of treatment for genetic or infectious
diseases using small, synthetic DNA-like molecules called AONs. Recent advances in the …