Kevin Campbell, PhD, on the Challenges of Gene Therapy Approaches in Advanced Muscular Dystrophy


The Howard Hughes Investigator at the University of Iowa discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.

“The big implication is that you can treat older animals with the gene transfer and it can be effective, but there's still work to be done in terms of making it fully effective for some integrated muscle physiology functions.”

As doctors and researchers have gained more experience with gene therapy as a modality in clinical trials and in the real world setting, it has become clear that earlier treatment with gene therapy with regard to the onset of disease symptoms typically leads to better outcomes. In other words, in young patients who receive a gene therapy product for a disease like muscular dystrophy prior to or shortly after the onset of symptoms, the product can serve as more of a preventative approach. On the other hand, older patients who receive gene therapy long after the progression of disease symptoms may experience some benefit, but will likely continue to suffer from disease complications that the gene therapy is unable to reverse. As such, continued research into disease pathogenesis is of great importance to understand and address the shortcomings of gene therapy approaches for these older patients.

Kevin Campbell, PhD, a Howard Hughes Investigator at the University of Iowa, gave a keynote speech on this topic at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024. In an interview with CGTLive® after his talk, Campbell discussed the details of his lab’s mouse model research into the pathogenesis of muscular dystrophy and the outcomes of treating older mice with gene therapy for muscular dystrophy. He noted that older mice that received gene therapy in a state of severe disease experienced some improvement in their condition, such as increases in weight and lifespan, but did not experience full recovery of grip strength and respiratory function. Campbell stated that the reason for this is not fully known and that as such more work is needed in order to understand how to overcome these shortcomings of the gene therapy modality.

Click here to view more coverage of the 2024 ASGCT Annual Meeting.

1. Campbell K. Mechanistic insights and therapeutic approaches to restore muscle function in muscular dystrophy. Presented at: ASGCT Annual Meeting 2024, May 7-10; Baltimore, Maryland.

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