Gene Therapy Enters Prime Time
October 25, 2020 | 10:30 AM - 12:30 PM
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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
Moderators
- Benjamin S. Freedman, PhD
- Fangming Lin, MD, PhD, FASN
Presentations
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Gene Therapy in Humans
10:30 AM - 11:00 AM
Gene Therapy in Humans
October 25, 2020 | 10:30 AM - 11:00 AM
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PiggyBac'ing Genes on the Kidney
11:00 AM - 11:30 AM
Matthew H. Wilson, MD, PhD
Matthew H. Wilson, MD, PhD
PiggyBac'ing Genes on the Kidney
October 25, 2020 | 11:00 AM - 11:30 AM
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Stem Cell Gene Therapy for Cystinosis
11:30 AM - 12:00 PM
Stephanie Cherqui, PhD
Stephanie Cherqui, PhD
Stephanie Cherqui is Aassociate Professor in the Department of Pediatrics, Division of Genetics at UC San Diego. Her lab specialized in the translation of gene therapy strategy from Bench-to-Bedside. She established that hematopoietic stem and progenitor cells (HSPCs) transplantation could reverse multi-organ degenerative disorders, even in the context of an intracellular membrane protein such as cystinosis. Her work led to the first in human HSPC gene therapy clinical trial for cystinosis. She is now applying this strategy to other disorders including the neuro-muscular degenerative disease, Friedreich’s ataxia.
Stem Cell Gene Therapy for Cystinosis
October 25, 2020 | 11:30 AM - 12:00 PM
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Gene Therapy for CKD
12:00 PM - 12:30 PM
Joseph V. Bonventre, MD, PhD, FASN
Joseph V. Bonventre, MD, PhD, FASN
Gene Therapy for CKD
October 25, 2020 | 12:00 PM - 12:30 PM
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