A CFTR mRNA isoform for the treatment of 3’ CFTR PTC variants with Normand Allaire
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A CFTR mRNA isoform for the treatment of 3’ CFTR PTC variants with Normand Allaire
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In this episode, Dr. Paloma Giangrande, Editor-in-Chief of Molecular Therapy Nucleic Acids, speaks with Normand Allaire, Head of Genomics at the Cystic Fibrosis Foundation Therapeutics Lab. They discuss the article, Upregulation of a CFTR mRNA isoform has therapeutic potential for the treatment of 3’ CFTR PTC variants, which introduces an ASO-based therapeutic approach to cystic fibrosis for the 10% of patients who lack an approved treatment.
Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au
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