ASN's Mission

ASN leads the fight to prevent, treat, and cure kidney diseases throughout the world by educating health professionals and scientists, advancing research and innovation, communicating new knowledge, and advocating for the highest quality care for patients.

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Kidney Week

Basic/Clinical Science Session

Gene Therapy Enters Prime Time

October 25, 2020 | 10:30 AM - 12:30 PM

Location: Live-Streamed

Session Description

Advances in gene therapy "vectorology" and molecular genetics have led to the first gene therapies in humans approved by the US Food and Drug Administration. Coupled with gene editing, this technology offers expanded opportunities for gene replacement or gene correction in inherited and acquired kidney diseases. This session reviews strategies for in vivo and ex vivo gene therapy and describes clinical trials of gene therapy for hemoglobinopathies and kidney diseases.

Learning Objective(s)

  • Explain the basis for gene therapy and therapeutic considerations
  • Describe recent clinical trials of gene therapy in humans
  • Identify advances in gene therapy for kidney diseases

Learning Pathway(s)

  • Genetic Diseases of the Kidneys
  • Development and Pediatrics


  • Benjamin S. Freedman, PhD
  • Fangming Lin, MD, PhD, FASN


  • Gene Therapy in Humans
    10:30 AM - 11:00 AM
    Edward J. Benz, MD
  • PiggyBac'ing Genes on the Kidney
    11:00 AM - 11:30 AM
    Matthew H. Wilson, MD, PhD
  • Stem Cell Gene Therapy for Cystinosis
    11:30 AM - 12:00 PM
    Stephanie Cherqui, PhD 
  • Gene Therapy for CKD
    12:00 PM - 12:30 PM
    Joseph V. Bonventre, MD, PhD, FASN