Volume 30, Issue 173, July 2026

The Future of DMD Therapy: A Systematic Review of CRISPR-Cas9 Innovations and Therapeutic Challenges

Monika Krasoń^1♦, Julia Witkowska¹, Ksenia Jakubiak¹, Krystian Woźniak¹, Adam Brożyna², Agnieszka Mackiewicz³, Aleksandra Pietrucień³, Natalia Kursa¹, Katarzyna Bielak⁴, Mikołaj Kurczyński¹

¹Central Teaching Hospital of The Medical University of Lodz: Łódź, 92-213, ul. Pomorska 251 Poland
²University Clinical Hospital No. 1 of the Medical University of Lodz: Łódź, 90-153, ul. Kopcińskiego 22, Poland
³Medical Centre of Pabianice, Pabianice, 95-200, ul. Jana Pawła II 68, Poland
⁴5 Military Clinical Hospital in Cracow, 30-901 Cracow, ul. Wrocławska 1-3, Poland

^♦Corresponding author
Monika Krasoń, address: Giedlarowa 1079; 37-300 Leżajsk, Poland

ABSTRACT

Duchenne muscular dystrophy (DMD) is a rare progressive neuromuscular disorder caused by an absence of dystrophin that results in generalized muscle weakness and muscle wasting. DMD is inherited in an X-linked manner and results in irreversible loss of muscle tissue and early death. Standard treatment includes corticosteroids and palliative care, which help manage some symptoms without treating the actual genetic defect. CRISPR-Cas9 genome editing is a rapidly advancing area of neuromuscular research, with the potential to permanently fix the underlying genetic cause at the DNA level. In preclinical studies it has been shown that CRISPR technology can restore dystrophin through the use of methods such as exon skipping or frame-restoration, and thus decrease tissue degeneration due to dystrophin deficit. New advances in base and prime editing provide more precise methods for genetic correction and less likelihood of double-strand breaks than some previous-generation "cut-and-paste" gene-editing strategies. Nevertheless, shifting these methods from animal models to human therapies brings significant challenges. Considerations include the immune response to adeno-associated virus (AAV) vectors, the potential for off-target effects, and challenges with delivering the treatment to all muscle groups, most notably the heart joint and diaphragm. New clinical research, including the EMBARK clinical trial results, shows how difficult it is for patients to achieve long-lasting benefits. Despite these challenges, continued improvement in the accuracy of gene editing presents some hope that DMD will have an opportunity to be transitioned from a symptom management to a cure for the disease if current safety and delivery challenges can be overcome.

Keywords: Duchenne muscular dystrophy, CRISPR Cas9 genome editing, dystrophin restoration

Medical Science, 2026, 30, e125ms3899

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Published: 18 July 2026