Revolutionizing Treatment For Duchenne Muscular Dystrophy with Gene Editing: Courtney Young, PhD

WATCH TIME: 3 minutes

“In general, the idea of gene therapy being able to revolutionize disease, both for root gene replacement, but other ways of delivering other things [is particularly exciting for me]. Using gene therapy to deliver CRISPR and using it to deliver ways to inactivate proteins to replace larger genes by creating overlapping sequences that can recombine. I think the idea of targeting the underlying cause of disease is what's going to be revolutionary for the field.”

Researchers are in the development process of creating gene editing therapies for neuromuscular diseases that are impacted by gene mutations. For example, MyoGene Bio’s MyoDys45-55 is a preclinical gene editing therapy that targets the DNA region where mutations are found in approximately half of patients with Duchenne muscular dystrophy (DMD). Gene editing, the next possible generation of gene therapy, is advantageous in that it has the ability to make changes in DNA that could retain more of the patient's protein coding sequence. Although gene therapy is relatively a new approach, it has shown its potential as a treatment for other neuromuscular diseases such as spinal muscular atrophy and may be promising approach to revolutionize the neuromuscular field.¹

At the recently concluded 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas, Courtney Young, PhD, the co-founder and chief executive officer of MyoGene Bio, presented data on the company’s mouse model research on MyoDys45-55 in a talk on the translation of gene editing technologies for clinical practice. The rest of the session focused on the potential of using CRISPR as a tool to correct genetic mutations in neuromuscular diseases and the challenges associated with developing effective gene editing therapies. Speakers presented the latest scientific data with gene editing platforms used in preclinical models for neuromuscular diseases.¹

In an interview with CGTLive®, a sister publication to NeurologyLive®, Young provided an overview of the talk session and the discussions around gene editing therapies. In addition, she spoke about the potential advantages of gene editing in clinical trials and clinical care for patients. Young also shared her thoughts on some of the presentations that stood out to her as a topic of interest and that she finds particularly exciting to her in the field.

Click here for more coverage of MDA 2023.

REFERENCES
1. Kemaladewi D, Young C, Rashnonejad A. Upregulation of LAMA1 as a Mutation-Independent Therapeutic Approach for LAMA2-CMD. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Translation of Gene Editing Technologies session.

Editor’s Note: Young disclosed that she is an owner and board member of MyoGene Bio, chief executive officer of MyoGene Bio, an inventor of patents belonging to MyoGene Bio and UCLA, an inventor of patents belonging to UCLA, and that she has stock ownership in MyoGene Bio and Skygene (startups developing therapies for muscle diseases).