In recent years, with the advent of sequencing technology, the diagnosis is no longer the major issue for Duchenne/Becker muscular dystrophy (DMD/BMD). The research hotspot for these disorders was therefore transferred to the treatment due to the lack of a definitive cure. In addition to the use of steroids and other compounds, new strategies such as stem cell or gene therapy are highlighted, which might be able to cure these disorders. However, these novel approaches still face major unsolved issues that hinder their clinical translation.
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
In recent years, with the advent of sequencing technology, the diagnosis is no longer the major issue for Duchenne/Becker muscular dystrophy (DMD/BMD). The research hotspot for these disorders was therefore transferred to the treatment due to the lack of a definitive cure. In addition to the use of steroids and other compounds, new strategies such as stem cell or gene therapy are highlighted, which might be able to cure these disorders. However, these novel approaches still face major unsolved issues that hinder their clinical translation.
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
