Gene Therapy: Cultivating Cures

Presenter: Rebecca Kaviani, PA-C

This presentation provides a comprehensive overview of emerging gene therapy treatments for patients with sickle cell disease. Participants will explore the pathophysiology of sickle cell disease, limitations of traditional management strategies, and the clinical application of recently approved gene therapy products. Emphasis is placed on patient selection, treatment processes, nursing responsibilities, and long-term monitoring.

The session reviews autologous stem cell collection, ex vivo gene modification, conditioning regimens, infusion procedures, and post-treatment follow-up. Nursing roles in patient education, symptom management, and psychosocial support are highlighted throughout the care continuum.

Learning Objectives:

Upon completion of this activity, participants will be able to:

• Describe the pathophysiology and clinical impact of sickle cell disease
• Explain current gene therapy approaches for sickle cell treatment
• Identify key steps in the gene therapy treatment process
• Discuss nursing responsibilities before, during, and after therapy
• Recognize potential risks, complications, and long-term outcomes

Target Audience: Registered nurses, advanced practice providers, transplant nurses, and healthcare professionals involved in hematology and cellular therapy.