Related Experiment Video
Updated: Jun 18, 2025
CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
Published on: September 14, 2019
Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy
Heidi Cope1, Ryan Fischer2, Emma Heslop3
1RTI Health Solutions, Research Triangle Park, NC, USA.
Background:
Duchenne muscular dystrophy (DMD) is a progressive, life-limiting, neuromuscular disorder. Clinicians play an important role in informing families about therapy options, including approved gene therapies and clinical trials of unapproved therapies.
Objective:
This study aimed to understand the perspectives of clinicians about gene therapy for DMD, which has not previously been studied.
Methods:
We conducted interviews with specialist clinicians treating patients with DMD in the United States (n = 8) and United Kingdom (n = 8). Interviews were completed in 2022, before any approved gene therapies, to gain insight into barriers and facilitators to implementing gene therapy and educational needs of clinicians.
Results:
Most respondents expressed cautious optimism about gene therapy. Responses varied regarding potential benefits with most expecting delayed progression and duration of benefit (1 year to lifelong). Concern about anticipated risks also varied; types of anticipated risks included immunological reactions, liver toxicity, and cardiac or renal dysfunction. Clinicians generally, but not uniformly, understood that gene therapy for DMD would not be curative. Most reported needing demonstrable clinical benefit to justify treatment-related risks.
Conclusions:
Our data demonstrate variability in knowledge and attitudes about gene therapy among clinicians who follow patients with DMD. As our knowledge base about DMD gene therapy grows, clinician education is vital to ensuring that accurate information is communicated to patients and families.
Insights
Clinician perspectives on gene therapy for Duchenne muscular dystrophy (DMD) show cautious optimism but highlight varied knowledge and educational needs. Ensuring accurate information is key for families considering these advanced DMD treatments.
Area of Science:
- Neuromuscular Disorders
- Gene Therapy
- Clinical Practice
Background:
- Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disease.
- Clinicians are crucial in guiding families on treatment options, including gene therapies and clinical trials.
Purpose of the Study:
- To explore U.S. and U.K. clinicians' views on gene therapy for DMD.
- To identify barriers, facilitators, and educational needs for implementing DMD gene therapy.
Main Methods:
- Semi-structured interviews were conducted with 16 specialist clinicians (8 U.S., 8 U.K.) in 2022.
- The study focused on perspectives before the approval of any gene therapies for DMD.
Main Results:
- Most clinicians expressed cautious optimism regarding gene therapy for DMD.
- Anticipated benefits and risks (e.g., immunological, organ toxicity) varied among respondents.
- Clinicians generally understood gene therapy is not curative but requires demonstrable benefit to outweigh risks.
Conclusions:
- Significant variability exists in clinician knowledge and attitudes toward DMD gene therapy.
- Ongoing clinician education is essential for disseminating accurate information to patients and families regarding DMD gene therapy.
Related Concept Videos
00:59Gene Therapy
01:21Satellite Stem Cells and Muscular Dystrophy
00:49What is Genetic Engineering?
01:20Myasthenia Gravis: Overview and Treatment
These antibodies interfere with the function of the nicotinic receptors in three ways: by binding to the receptor and disrupting acetylcholine binding; by causing cross-linking of receptors which...
01:59CRISPR
02:59Animal Mitochondrial Genetics