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

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&rsquo; CFTR PTC variants, which introduces an ASO-based therapeutic approach to cystic fibrosis for the 10% of patients who lack an approved treatment.
Music:&nbsp;'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.&nbsp;<a href="https://www.scottbuckley.com.au/">www.scottbuckley.com.au</a>

The American Society of Gene & Cell Therapy is the premier membership organization for professionals working in cell and gene therapy. On this network, we're sharing exciting stories from our members and the greater scientific community.

The American Society of Gene &amp; Cell Therapy is the premier membership organization for professionals working in cell and gene therapy. On this network, we're sharing exciting stories from our members and the greater scientific community.

ASGCT Podcast Network

A CFTR mRNA isoform for the treatment of 3’ CFTR PTC variants with Normand Allaire

This episode, recorded at the ASGCT 2026 Annual Meeting in Boston, focuses on Molecular Therapy Advances’ upcoming special issue, Beyond prevalance: How rare diseases are reshaping therapeutic innovation. Listen as Editor-in-Chief of Molecular Therapy Advances, Dr. Mohamed Abou-el-Enein, discusses the evolution of therapeutic development for rare diseases with guest editors Drs. Jennifer Adair, Claire Booth, Terence Flotte, and Donald Kohn.

Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

This episode, recorded at the ASGCT 2026 Annual Meeting in Boston, focuses on&nbsp;Molecular Therapy Advances&rsquo; upcoming special issue, <a href="https://www.cell.com/molecular-therapy-family/advances/special-issues/call-for-papers/rare-diseases">Beyond prevalance: How rare diseases are reshaping therapeutic innovation</a>.&nbsp;Listen as Editor-in-Chief of&nbsp;Molecular Therapy Advances, Dr. Mohamed Abou-el-Enein, discusses the evolution of therapeutic development for rare diseases with guest editors Drs.&nbsp;Jennifer Adair, Claire Booth, Terence Flotte, and Donald Kohn.
Music:&nbsp;'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.&nbsp;<a href="https://www.scottbuckley.com.au/">www.scottbuckley.com.au</a>

How rare diseases are shaping therapeutic innovation with Jennifer Adair, Claire Booth,Terry Flotte and Donald Kohn

Scientists, Unedited is a monthly podcast hosted by Eirini Vamva, PhD, that spotlights the people behind scientific breakthroughs – their journeys, motivations, and lives beyond the lab. Each episode begins with personal stories, explores passions outside of science, and closes with an accessible conversation about the guest’s work for a general audience.

In this first episode, Dr. Vamva sits down with ASGCT President Matthew Porteus, PhD, for a conversation about the winding path that brought him full circle back to Stanford University. The conversation offers a candid look at the personal experiences and motivations that have shaped Dr. Porteus’s career and his leadership in the field of cell and gene therapy.

Scientists, Unedited is a monthly podcast hosted by Eirini Vamva, PhD, that spotlights the people behind scientific breakthroughs &ndash; their journeys, motivations, and lives beyond the lab. Each episode begins with personal stories, explores passions outside of science, and closes with an accessible conversation about the guest&rsquo;s work for a general audience.
In this first episode, Dr. Vamva sits down with ASGCT President Matthew Porteus, PhD, for a conversation about the winding path that brought him full circle back to Stanford University. The conversation offers a candid look at the personal experiences and motivations that have shaped Dr. Porteus&rsquo;s career and his leadership in the field of cell and gene therapy.

A Full-Circle Journey at Stanford: Get to Know ASGCT President Matthew Porteus, PhD

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini-series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community.
 
In this episode, host Ben McLeod of the ASGCT Communications Committee talks with Godwin Iroanya of the University of Missouri. Godwin shares his current work and research and how ASGCT membership has helped his career, including the Molecular Therapy Family of Journals and the Society’s pilot Mentor Program.

Music: 'Origami' by Scott Buckley – released under CC-BY 4.0. www.scottbuckley.com.au

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini-series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community. &nbsp; In this episode, host Ben McLeod of the ASGCT Communications Committee talks with Godwin Iroanya of the University of Missouri. Godwin shares his current work and research and how ASGCT membership has helped his career, including the Molecular Therapy Family of Journals and the Society&rsquo;s pilot Mentor Program.
Music:&nbsp;'Origami' by Scott Buckley &ndash; released under CC-BY 4.0.&nbsp;<a href="https://url.us.m.mimecastprotect.com/s/IkczCERmXJSyRj4iNfxU7G5vB">www.scottbuckley.com.au</a>

A CFTR mRNA isoform for the treatment of 3’ CFTR PTC variants with Normand Allaire

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