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Myofilament Targeted Gene Therapy

Heart and skeletal muscle diseases are a significant healthcare concern, often leading to progressive loss of mobility, cardiovascular function and death. Indeed, heart failure is a growing epidemic and is now the number one killer worldwide while muscle diseases such as muscular dystrophy are a main cause of morbidity and mortality in young males. However, the current standard of care for most muscle disease is merely palliative and primarily provides management of muscle degeneration and loss of function. New treatments are needed to slow, halt or even reverse this progression and to improve the quality of life for afflicted individuals. Approaches such as genetic and cellular engineering have great potential for improving many forms of both skeletal and heart muscle disease. Recent advances in the development of these approaches, and in gene delivery methods, have now made gene therapy a viable approach for clinical development and treatment of muscle diseases.

This Special Issue of Regenerative Medicine Frontiers will be devoted to gene therapy targeted to the contractile organelles of muscle cells, the myofibril. Several contractile and regulatory proteins of the myofilaments in muscle cells, and proteins such as enzymes that modify their behavior, that are potential targets for gene therapy. In this Special Issue we are looking for original research papers, short reviews or commentaries on this approach to treat cardiac and skeletal muscle diseases.

Professor Michael Regnier

Guest Editor

Keywords

gene therapy

muscle

myofilament

contractile proteins

engineered protein

AAV

myopathy

Submission Deadline: 31 October 2019

Online Submission

Manuscripts should be submitted online through Hapres Online Submission System. Please visit Guide for Authors before submitting a manuscript. Authors are encouraged to submit a paper as soon as it is ready and don’t need to wait until the deadline. Submissions will be sent to peer-review in order of arrival. Accepted papers will be published continuously in Regenerative Medicine Frontiers (RMF) and then gathered together on the special issue webpage. We welcome Research articles, Review papers and Short Communications. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.



Note

Virtual Special Issue (VSI) is a collection of papers centered around a specific topic, led by an expert (Guest Editor) in the field. Virtual Special Issues are an important component of our journal and cover current hot topics within the scope of the journal.

All papers belonging to a Virtual Special Issue will be gathered together on a single webpage. They are published in the regular issues of the journal as soon as publishable, and labeled as belonging to the Virtual Special Issue. A link from each paper will take you to the Virtual Special Issue website.

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Guest Editor

  • Professor Michael Regnier

    Washington Research Foundation Endowed Professor of Bioengineering; Vice Chair for Research, Bioengineering
    University of Washington, Seattle, USA

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