The CFRI CF Community Voices Podcast

Nanotechnologies to Enable Cystic Fibrosis Gene Therapies – Steven Jonas, MD, PhD

Dr. Steven Jonas, a pediatric physician scientist and Assistant Professor in the Department of Pediatrics at the UCLA David Geffen School of Medicine and California NanoSystems Institute, presents results from CFRI-funded research on nanotechnologies to enable cystic fibrosis gene therapies. He highlights his team’s approach to overcome common obstacles to gene therapy through the design and testing of CRISPR/Cas9-based gene-editing cargoes for CFTR correction and nanoparticle carriers that can be delivered as aerosolized suspensions to enable inhalable gene therapy solutions for CF. This presentation was part of CFRI's Virtual 35th National CF Education Conference.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Selective Targeting Distal Small Airways by AAV Vectors (...) – Xiaopeng Li, PhD

Dr. Xiaopeng Li, Associate Professor in the Department of Pediatrics and Human Development in the College of Human Medicine at Michigan State University, presents results from CFRI-funded research on selective targeting distal small airways by adeno-associated virus (AAV) vectors. He discusses findings from CF pig models that support AAV4 as a suitable viral vector for CF gene therapy to target small airway secretory cells. This presentation was part of CFRI's Virtual 35th National CF Education Conference.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

The Challenges of Aging for People with CF - Richard Simon, MD (Audio)

Dr. Richard Simon, Professor Emeritus at the Division of Pulmonary and Critical Care Medicine at the University of Michigan, gives a comprehensive overview of the health challenges faced by an aging CF population. In addition to general aging symptoms such as decreasing mobility, reduced strength, and memory loss, there are CF-specific issues such as CF-related diabetes, CF-related bone disease, and gastrointestinal cancers. This presentation was part of CFRI's Virtual 35th National CF Education Conference on July 30, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Oral Health Considerations for Individuals with CF – Donald Chi, DDS, PhD (Audio)

Dr. Donald Chi, Professor and the Lloyd and Kay Chapman Endowed Chair of Oral Health at the University of Washington School of Dentistry in Seattle, WA, gives a comprehensive summary of the available scientific data on the oral health of individuals with CF. He presents information on CF-specific risk factors that can lead to poor oral health and provides information about ongoing studies aimed at understanding the links between oral and overall health in CF. This presentation was part of CFRI's Virtual 35th National CF Education Conference.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Novel Models of CF Mucus Plugs for Testing Phage and Tailocin Therapy – Gregory Burkeen, PhD Candidate (Audio)

Gregory Burkeen, a PhD Candidate at San Diego State University in Drs. Anca Segall and Forest Rohwer’s labs, presents results from CFRI-funded research on novel models of CF mucus plugs for testing phage and tailocin therapy. He proposes an in-vitro model based on artificial sputum that is tailored to the individual, and can be used to test the safety and efficacy of personalized therapeutic regimens. This presentation was part of CFRI's Virtual 35th National CF Education Conference.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Nanotechnologies to Enable Cystic Fibrosis Gene Therapies – Steven Jonas, MD, PhD (Audio)

Dr. Steven Jonas, a pediatric physician scientist and Assistant Professor in the Department of Pediatrics at the UCLA David Geffen School of Medicine and California NanoSystems Institute, presents results from CFRI-funded research on nanotechnologies to enable cystic fibrosis gene therapies. He highlights his team’s approach to overcome common obstacles to gene therapy through the design and testing of CRISPR/Cas9-based gene-editing cargoes for CFTR correction and nanoparticle carriers that can be delivered as aerosolized suspensions to enable inhalable gene therapy solutions for CF. This presentation was part of CFRI's Virtual 35th National CF Education Conference.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Selective Targeting Distal Small Airways by AAV Vectors (...) – Xiaopeng Li, PhD (Audio)

Dr. Xiaopeng Li, Associate Professor in the Department of Pediatrics and Human Development in the College of Human Medicine at Michigan State University, presents results from CFRI-funded research on selective targeting distal small airways by adeno-associated virus (AAV) vectors. He discusses findings from CF pig models that support AAV4 as a suitable viral vector for CF gene therapy to target small airway secretory cells. This presentation was part of CFRI's Virtual 35th National CF Education Conference.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Joints in Cystic Fibrosis - Dr. Beth Clarke

Many individuals with cystic fibrosis suffer from chronic joint pain. In this podcast, recorded at CFRI's CF Adult Retreat in August, 2022, Dr. Beth Clarke, from the University of Glasgow in Scotland, discusses the causes of joint pain and cystic fibrosis associated arthritis (CFA), exercise and CFA and strategies to address this pain.

If you watch this episode, please let us know what you think at https://www.surveymonkey.com/r/KGB7PKY

CF Community Voices is made possible through grants from Chiesi USA, Genentech, Vertex Pharmaceuticals, Gilead Sciences, and Viatris.

CFRI’s 35th Cystic Fibrosis Education Conference - Awards Celebration on July 30, 2022

At CFRI's virtual Conference Awards Celebration on July 30, 2022, we honored these extraordinary individuals: Richard Moss, MD - Paul M. Quinton Cystic Fibrosis Research Legacy Award; Sonya Haggett, LCSW - CFRI Partners in Living Award In Memory of Anabel Stenzel; Anna Payne – Dave Stuckert Memorial Volunteer of the Year Award; Dennis Nielson, MD, PhD - Professional of the Year Award.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Keynote Remarks by Janet Woodcock, MD - CFRI’s 35th Cystic Fibrosis Education Conference

Dr. Janet Woodcock, Principal Deputy Commissioner of the US Food and Drug Administration, delivered Keynote Remarks at the opening of CFRI’s 35^th National CF Education Conference on July 30, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.