About this Research Topic
For stem cell therapy, further improvement of engraftment efficiency is the key point for further clinical translation. For gene therapy, there is a need for longevity of dystrophin expression, along with the safety concerns related to the gene therapy approach. In addition, muscle stem cells (satellite cells) are refractory to current gene therapy methods.
In the meantime, the precision diagnosis for DMD/BMD is still not a trivial task. For example, distinguishing DMD from BMD during infancy and toddlerhood is important to choose the treatment for patients, and considering that some potential treatments are underway, screening is also helpful for early diagnosis and treatment.
While the studies about precision diagnostics and treatment for DMD/BMD are ongoing, there is no systematic work that analyzes the latest development. This collection focuses on novel research about precision diagnostics and treatment for DMD/BMD and we welcome submissions of Original Research, Reviews, and other article types (excluding case reports) on the following themes:
(1) Effective newborn screening strategies for DMD/BMD
(2) Novel approaches for a precision diagnosis of DMD/BMD during infancy and toddlerhood
(3) New biomarkers for monitoring disease progression and treatment response
(4) Recent advances in stem cell therapy and gene therapy for DMD/BMD
(5) Other novel treatments for DMD/BMD
Keywords: Duchenne/Becher Muscular Dystrophy, DMD, BMD, Precision Diagnostic, treatment
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