Research Topic

Gene Therapy for Neuromuscular Diseases

About this Research Topic

For a long time, the treatment of neuromuscular diseases has remained substantially symptomatic. However, thanks to the advent of new technologies, over the last few years there has been a constant growth in therapeutic studies and clinical trials in the field of neuromuscular diseases.

This has led to the possibility of applying innovative therapeutic methods such as gene replacement, gene editing, and antisense oligonucleotide therapy, to specific neuromuscular diseases. In particular, some of the diseases for which these therapies have been developed include spinal muscular atrophy and muscular dystrophies.

This Research Topic aims to collect high-quality manuscripts detailing the most recent advances in the use of gene therapy for the treatment of inherited neuromuscular diseases. With this Research Topic we aim to obtain a comprehensive update on current applications and future prospects of this new therapeutic strategy.


Keywords: Gene Replacement Therapy, Gene Editing, AAV, Antisense Oligonucleotide Therapy, Exon Skipping


Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.

For a long time, the treatment of neuromuscular diseases has remained substantially symptomatic. However, thanks to the advent of new technologies, over the last few years there has been a constant growth in therapeutic studies and clinical trials in the field of neuromuscular diseases.

This has led to the possibility of applying innovative therapeutic methods such as gene replacement, gene editing, and antisense oligonucleotide therapy, to specific neuromuscular diseases. In particular, some of the diseases for which these therapies have been developed include spinal muscular atrophy and muscular dystrophies.

This Research Topic aims to collect high-quality manuscripts detailing the most recent advances in the use of gene therapy for the treatment of inherited neuromuscular diseases. With this Research Topic we aim to obtain a comprehensive update on current applications and future prospects of this new therapeutic strategy.


Keywords: Gene Replacement Therapy, Gene Editing, AAV, Antisense Oligonucleotide Therapy, Exon Skipping


Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.

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Submission Deadlines

13 December 2020 Abstract
12 April 2021 Manuscript

Participating Journals

Manuscripts can be submitted to this Research Topic via the following journals:

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Topic Editors

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Submission Deadlines

13 December 2020 Abstract
12 April 2021 Manuscript

Participating Journals

Manuscripts can be submitted to this Research Topic via the following journals:

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