Introduction to Gene Therapy: Key Terms, Concepts, and International Perspectives - Annual Meeting 2019

ACPE UAN #:  0233-0000-19-013-H04-P

Release Date:  March 25, 2019

Expiration Date:   March 25, 2022

Activity Type:    Knowledge-based

Contact Hours:  1 

Activity Fee:  Member:  $15        Non-Member:  $30

Target Audience: Pharmacists who practice in managed care settings.

Activity Description

Gene therapy is an investigational technique that offers the exciting promise of curing many debilitating and potentially terminal diseases, such as cystic fibrosis, multiple sclerosis, hemophilia, sickle cell disease, and spinal muscular atrophy. Rather than treating these conditions with medications or surgical interventions, gene therapy may allow disorders to be treated by inserting a gene into a patient’s DNA to alter gene expression. Methods to modify DNA include replacing a mutated gene associated with a disease with a healthy copy of the gene, inactivating, or “knocking out,” a mutated gene that is functioning improperly or introducing a new gene that can help fight a disease. Although gene therapy holds great potential for improving the treatment of many conditions, there are many challenges that must be overcome to develop treatments that are safe and effective. Identification of effective vectors (e.g., inactivated viruses, bacteria, liposomes, and plasmids) that will insert a gene into DNA is a critical step. In addition, it is important to ensure that gene therapy does not trigger a harmful immune response.

This session will provide an overview of key terms and concepts of gene therapy, while also providing an international perspective.

Learning Objectives:  

At the completion of this activity, participants should be able to:

  1. Explain the key terms and concepts of gene therapy.
  2. Describe investigational gene therapy strategies and how they may influence the manifestations of various disorders.
  3. Compare and contrast various types of vectors used in the delivery of gene therapy.
  4. Describe the experience of gene therapies internationally. 

Schedule of Educational Activity

This activity consists of an audio recording, pre-test assessment questions (not required to pass or continue), post-test questions (required to pass with a score of 70% or higher) and a specific activity evaluation. 

The Academy of Managed Care Pharmacy (AMCP) is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education.

As an accredited provider, AMCP strictly adheres to the ACPE Standards for Commercial Support and other relevant guidelines and standards to ensure the independence of continuing pharmacy education (CPE) activities.  It is AMCP’s intent that all CPE activities be independent, objective, balanced, evidence-based and free of any commercial bias.

CPE Requirements:   

Participants should claim continuing pharmacy education (CPE) credit for this home study activity only if they have not claimed credit for the live activity presented at Specialty Connect 2019.

CPE credit will be processed through the AMCP Learn portal and reported directly to CPE Monitor.  Please allow 72 hours for processing and posting in your NABP account.

To receive credit for an online activity you must:

  • Review the full content of the activity.
  • Successfully complete the post-test (required to pass with a score of 70% or higher, it may be taken a maximum of three times).
  • Complete a specific activity evaluation.

Statement of Commercial Support

This educational activity is supported by independent educational grants from AveXis Inc., Genzyme Corporation, Pfizer and Sarepta Therapeutics.


Clifford Goodman, PhD | Senior Vice President & Director | Lewin Center for Comparative Effectiveness Research | Falls Church, Virginia.

System Technical Requirements/Viewing Requirements: System Requirements Courses and learning activities are delivered via your Web browser and Acrobat PDF. For all activities, you should have a basic comfort level using a computer and navigating websites.  View the minimum technical and system requirements for continuing education online learning activities.

Credit Information

Activity Number Credit Amount Accreditation Period
ACPE UAN #0233-0000-19-013-H04-P 1 Contact Hour March 25, 2019 - March 25, 2022