Molecular-targeted therapy for Duchenne muscular dystrophy: progress and potential

Area of Science:

• Genetics
• Neurology
• Regenerative Medicine

Background:

• Duchenne muscular dystrophy (DMD) is a severe X-linked genetic disorder causing progressive muscle degeneration.
• Advances in multidisciplinary care have extended patient lifespan, yet a cure is not yet available.
• Current treatments do not prevent progressive disability or premature death in adulthood.

Purpose of the Study:

• To review the current state of gene and cellular therapy for Duchenne muscular dystrophy.
• To highlight the progress and challenges in developing curative treatments for DMD.
• To identify future research directions for managing and curing DMD.

Main Methods:

• Review of current viral and nonviral gene therapy strategies for DMD.
• Analysis of adeno-associated virus (AAV) vectors, naked plasmid DNA, antisense oligonucleotides, and gene editing techniques.
• Evaluation of research transitioning from preclinical to clinical settings.

Main Results:

• Gene and cellular therapies have advanced significantly, moving into clinical trials.
• Various sophisticated gene therapy approaches are under investigation for DMD.
• Despite progress, existing therapies still have limitations and require further development.

Conclusions:

• Cellular and gene therapies represent the most promising avenue for a Duchenne muscular dystrophy cure.
• Continued research is essential to overcome current therapeutic challenges and develop effective strategies.
• The ultimate goal is to devise treatments that can manage and potentially cure DMD, improving long-term outcomes.