A Platform for Gene Therapy Delivery: Adeno-Associated Virus (AAV) Vectors

Released On
September 8, 2021

Expires On
September 8, 2022

Media Type

Completion Time
45 minutes

Genetics, Hepatology, Neonatology, Nephrology, Pathology, Pediatrics

Genetic Disease, Neonatal

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This activity is provided by EXCEL Continuing Education.


This activity is supported by an independent education grant from ULTRAGENYX PHARMACEUTICAL.

Credit Available

  • Physicians — maximum of 0.75 AMA PRA Category 1 Credit(s)

All other healthcare professionals completing this course will be issued a statement of participation.

Target Audience

This activity is designed to meet the educational needs of geneticists, nephrologists, hepatologists, pediatricians, neonatologists, pathologists, and other HCPs who often contribute to the multidisciplinary team when managing patients with genetic disorders.

Program Overview

The goal of gene therapy is to treat diseases at the genetic level by replacing deficient enzymes in target tissues, therefore, changing the course of the disease. Simply stated, it is the transfer of genetic material for disease therapy or prevention. Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-term efficacy in several trials across target organs, including eye, liver, skeletal muscle, and the central nervous system.

The goal of this CME-certified activity is to increase physicians' knowledge of clinical considerations relating to the use of adeno-associated virus (AAV) vectors in gene therapy, and its impact on the treatment of several rare disorders.

Learning Objectives

Upon completion of this activity, participants should be better able to:

  • Analyze geneā€based treatment approaches that have the potential to change the disease course for patients with genetic disorders
  • Review the characteristics of AAV that allow it to be used as a vector for gene therapy
  • Evaluate evidence for the use of gene therapies, including AAV-based, that are approved or pending approval for the management of rare disorders
  • Counsel patients about clinical-trial opportunities



Eric T. Rush, MD, FAAP, FACMG

Clinical Geneticist
Underdown/Yeomans Family Endowed Professor in Connective Tissue Disorders Care
Medical Director, Office of Faculty Development
Children’s Mercy Kansas City
Associate Professor of Pediatrics
University of Missouri – Kansas City School of Medicine
Clinical Associate Professor of Internal Medicine
University of Kansas School of Medicine

Physician Accreditation Statement

EXCEL Continuing Education is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians.

Physician Credit Designation

EXCEL Continuing Education designates this enduring activity for a maximum of .75 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Disclosures of Conflicts of Interest

Eric T. Rush, MD

  • Consultant: Alexion Pharmaceuticals, Inozyme Pharma, Ultragenyx Pharmaceutical and Biomarin Pharmaceuticals
  • Speaker: Alexion Pharmaceuticals, Ultragenyx Pharmaceutical and Biomarin Pharmaceuticals
  • Research: Alexion Pharmaceuticals
  • Drug Monitoring Board: Ascendis, SA
  • Stock Ownership: Intercept Pharmaceuticals and Specturm Pharmaceuticals

EXCEL staff and Peer Reviewer: Nothing to disclose.

Instructions for Participation and Credit

There are no fees for participating and receiving CME credit for this enduring activity. To receive CME credit participants must:

  1. Read the CME/CE information and faculty disclosures.
  2. Complete pre-test
  3. Participate in the online activity.
  4. Complete post-test
  5. Submit the evaluation form.
Certificates will be emailed to the participant

Course Viewing Requirements

Supported Browsers:
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above

Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above

Disclosure of Unlabeled Use

EXCEL Continuing Education requires that faculty participating in any CME activity disclose to the audience when discussing any unlabeled or investigational use of any commercial product or device not yet approved for use in the United States.


This activity is designed for educational purposes. Participants have a responsibility to utilize this information to enhance their professional development to improve patient outcomes. Conclusions drawn by the participants should be derived from careful consideration of all available scientific information. The participant should use his/her clinical judgment, knowledge, experience, and diagnostic decision-making before applying any information, whether provided here or by others, for any professional use.

Contact Information

For CME questions please contact: info@excelcme.com