Title
Gene Therapy for Providers 101
Release Date
July 01, 2022
Expiration Date
The estimated time to complete this activity is 45 minutes.
Purpose Statement
The purpose of this activity is to enable learners to develop basic knowledge of gene therapy for hemophilia, thereby enhancing learner competence to discuss gene therapy with patients who may benefit from this emerging treatment.
Activity Description
This learning activity provides basic education on hemophilia gene therapy, including the aims of gene therapy; the packaging, delivery, and activity of the replacement genes; and the means by which the replacement genes lead to production and/or availability of normal clotting factor in persons with hemophilia. The module will also review information that may be particularly important when discussing hemophilia gene therapy with patients, such as knowledgeable informed consent, outcomes data, and potential risks and benefits.
Target Audience
This enduring material is intended for multidisciplinary providers at hemophilia treatment centers, including nurses, physicians, pharmacists, physical therapists, social workers, and genetic counselors providing care to persons with hemophilia A and hemophilia B.
Educational Objectives
At the conclusion of this enduring material, the participants should be better able to:
- Describe a pathway to preparedness for gene therapy from education of the multidisciplinary healthcare team to clinical delivery
- Provide an overview of the goal of gene therapy for hemophilia
- Explain the elements of gene therapy (i.e. packaging, administration, mechanism of action)
- Define the profile of gene therapy candidates with FVIII deficiency and FIX deficiency
- Review known adverse effects from completed and ongoing clinical trials
- Articulate critical considerations for the multidisciplinary healthcare team in providing care for patients receiving gene therapy
- Discuss issues related to obtaining knowledgeable informed consent and counseling patients
Faculty
- Steven Pipe, MD
- Shelley Crary, MD, MSCS
Contact Information
System Requirements
- 2 gigabytes of RAM
- High-speed internet connection
- Internet browser (current version) Firefox, Safari, Chrome, or Explorer
- Cookies and JavaScript must be enabled in the browser