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Cystic Fibrosis:

Gene Therapy

Ellie Johnson, Claire Strauel, Sam Bennett, Ashley Nelson

Background

  • Disease that damages lungs and other organs

  • Caused by defective CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene

  • Test through sweat test and blood test

  • Early death

  • Quite common

  • Inherited

Background

Signs/Symptoms

Signs/Symptoms

  • Very salty tasting skin

  • Persistant cough (with phlegm)

  • Lung infections

  • Wheezing/shortness of breath

  • Poor growth and weight gain

  • Frequent greasy stool and difficult bowel movement

  • Nasal polyps

  • Chronic sinus infections

  • Enlarged fingertips and toes

  • Rectul prolapse

  • Male infertility

What is Gene

Therapy?

  • Modifies how a gene is expressed

  • Treats diseases using replacement, inactivation, and introduction

  • Two types: In Vivo and Ex Vivo

Gene Therapy

Overview

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy

Gene Therapies Used In CF

Gene Therapy in CF

  • integrating gene therapy
  • correct CFTR gene integrated into the genome.
  • non-integrating gene therapy
  • correct CFTR gene is placed in the cell, but does not integrate into the genome.
  • RNA therapy
  • RNA replaces or repairs the mutated mRNA.

Therapeutic Gene Editing in CF: CRISPR/Cas9

  • RNA/protein complex that corrects the genetic mutations themselves.
  • Three main components:

- "Guide" RNA

- "scissors" Cas9 nuclease

- template strand

  • Allows for regulation at transcriptional level.
  • Still being tested, could be years before this is used to treat humans with CF.

Helpful Video

https://www.youtube.com/watch?v=Sp774i6tdzE&t=9s

Side Effects

Side Effects of Gene Therapy directly related to CF

- Weight gain

- Neurological issues (dizziness)

- Pulmonary exacerbations

General Side Effects of Gene Therapy

- Increased Risk of Cancer

- Immune reaction

- May target wrong cell

- Infection

https://play.kahoot.it/v2/?quizId=370e4437-9c24-41f4-97a9-a6e8afd98e4e

Kahoot!

Sources

  • Carlon, Marianne, Vidović, Dragana, & Birket, Susan. (2017). Roadmap for an early gene therapy for cystic fibrosis airway disease. Prenatal Diagnosis, 37(12), 1181–1190.
  • “Gene Editing for Cystic Fibrosis: Cystic Fibrosis Foundation.” Gene Editing for Cystic Fibrosis | Cystic Fibrosis Foundation.
  • Center for Biologics Evaluation and Research. (2018). What is gene therapy? U.S. Food and Drug Administration. Retrieved November 16, 2021, from https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy.
  • Ziying Yan, Paul B McCray Jr, John F Engelhardt, Advances in gene therapy for cystic fibrosis

lung disease, Human Molecular Genetics, Volume 28, Issue R1, October 2019, Pages

R88–R94, https://doi.org/10.1093/hmg/ddz139

  • Skilton M, Krishan A, Patel S, Sinha IP, Southern KW. Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis. Cochrane Database of Systematic Reviews 2019, Issue 1. Art. No.: CD009841. DOI: 10.1002/14651858.CD009841.pub3.
  • Mayo Foundation for Medical Education and Research. (2017, December 29). Gene therapy. Mayo Clinic. Retrieved November 16, 2021, from https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619.

Sources

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