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Gene Therapy for Duchenne Muscular Dystrophy

Gene Therapy For Duchenne Muscular Dystrophy

Gene Therapy for
Duchenne Muscular Dystrophy

Gene therapy offers the prospect of long-term and potentially curative benefits to patients with a genetic disease, including Duchenne muscular dystrophy.

Today, three Duchenne gene therapies are making their way through clinical trials and interim results from those studies have been very encouraging. As more opportunities to participate in clinical trials develop, it becomes even more important for us to ensure we’re familiar with the basics of gene therapy.

This video, created by the American Society of Gene and Cell Therapy (ASGCT), does a great job explaining gene therapy. I also included a list of current Duchenne gene therapy clinical trials as well as a few links to additional resources offering information about gene therapy that I think you’ll find helpful.

If you have any questions about gene therapy or would like to share your thoughts on the topic, please visit rareCourage. We’d love to hear from you!

Clinical Trials of
Duchenne Gene Therapy

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

Sarepta Therapeutics
A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)

Solid Biosciences
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Additional Resources for
Information About Gene Therapy


A Guide to Gene Therapy

source: Global Genes

The goal of this toolkit is to provide rare patients and caregivers with an overview of gene therapy and why it is being considered as a possible treatment for many rare diseases. To make the content easier to connect with and more engaging there are Illustrations, interactive journal prompts, and trivia about genes and jeans.

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Gene Therapy Basics

source: American Society of Gene and Cell Therapy

The American Society of Gene & Cell Therapy is the primary professional membership organization for gene and cell therapy. The mission of ASGCT is to advance knowledge, awareness, and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease.

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What is Gene Therapy?

source: Pfizer

Gene therapy is a technology aimed at correcting or fixing a gene that may be defective. This exciting and potentially transformative area of research is focused on the development of potential treatments for monogenic diseases, or diseases that are caused by a defect in one gene.

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The Role of the Vector

source: Sarepta Therapeutics

A vector is the vehicle used to transport the transgene—the genetic material that will make the protein of interest—to the target cells. Selecting the right vector is critical for 2 main reasons: 1) The vector needs to be able to deliver the transgene specifically to muscle cells. 2) Pre-existing immunity to a viral vector can sabotage the therapy’s effectiveness.

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Why Gene Transfer?

source: Solid Biosciences

Gene transfer is an investigational approach that aims to significantly slow or even halt the progression of Duchenne muscular dystrophy (Duchenne) by delivering a modified yet functional dystrophin gene throughout the body. Scientists have been exploring the potential of using genes as medicine to treat disease for more than 35 years. Several recent advancements have made it possible to apply this technology to Duchenne. Gene transfer is potentially beneficial in Duchenne because it aims to address the root cause of the disease. While the risks and benefits of gene transfer in Duchenne still need to be evaluated in human clinical trials, early research is encouraging.

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