Genetic conditions that affect bones, muscle or skin are common. As these three tissues form the structural foundation of the human body, genetic impairment of anyone of them usually has impact on the whole organism. Therapeutic approaches faced a number of challenges, such as inappropriate biodistribution of pharmaceutic molecules or difficulties to target specific progenitor cells (throughout the body) in tissues capable of regeneration. Both in the musculoskeletal system and in the skin, drug-targeting hurdles have remained difficult to overcome.
Promising results are emerging from ongoing gene-addition trials for Duchenne muscular dystrophy as well as from trials addressing genetic bone defects, e.g. an enzyme replacement therapy for hypophosphatemia. This research topic will focus on how gene, cell and protein replacement strategies have been used to develop curative therapeutic approaches to genetic disorders of the musculoskeletal system and the skin. It will review pre-clinical research involving animal models of disease and describe what we have learned about the advantages and limitations of corrective gene transfer to tissues as different as bone and skin. Examples of disorders will be discussed where replacement of a missing protein actually represents a more promising treatment strategy than gene therapy attempts. Tissue-specific requirements for the timing of interventions shall be considered. Lastly, the research topic will deliberate recent clinical developments in the field.
We encourage authors to submit original research, ranging from basic to clinical translational studies, reviews, opinions and perspectives, and clinical trial protocols including but not limited to any one or more of the following themes:
a) targeted gene delivery to progenitor cells in muscle, bone and skin (satellite cells, mesoangioblasts, osteoblasts, epidermal stem cells and others)
b) gene addition, exon skipping, stop codon read-through and gene repair in muscle tissue – and the specific challenges of genetic diseases characterized by progressive muscle degeneration
c) time-limited protein/enzyme replacement to treat genetic developmental conditions of the musculoskeletal system or the skin
d) current clinical trials and horizons of gene, cell and protein replacement therapy of genetic muscle, bone and skin disorders
Genetic conditions that affect bones, muscle or skin are common. As these three tissues form the structural foundation of the human body, genetic impairment of anyone of them usually has impact on the whole organism. Therapeutic approaches faced a number of challenges, such as inappropriate biodistribution of pharmaceutic molecules or difficulties to target specific progenitor cells (throughout the body) in tissues capable of regeneration. Both in the musculoskeletal system and in the skin, drug-targeting hurdles have remained difficult to overcome.
Promising results are emerging from ongoing gene-addition trials for Duchenne muscular dystrophy as well as from trials addressing genetic bone defects, e.g. an enzyme replacement therapy for hypophosphatemia. This research topic will focus on how gene, cell and protein replacement strategies have been used to develop curative therapeutic approaches to genetic disorders of the musculoskeletal system and the skin. It will review pre-clinical research involving animal models of disease and describe what we have learned about the advantages and limitations of corrective gene transfer to tissues as different as bone and skin. Examples of disorders will be discussed where replacement of a missing protein actually represents a more promising treatment strategy than gene therapy attempts. Tissue-specific requirements for the timing of interventions shall be considered. Lastly, the research topic will deliberate recent clinical developments in the field.
We encourage authors to submit original research, ranging from basic to clinical translational studies, reviews, opinions and perspectives, and clinical trial protocols including but not limited to any one or more of the following themes:
a) targeted gene delivery to progenitor cells in muscle, bone and skin (satellite cells, mesoangioblasts, osteoblasts, epidermal stem cells and others)
b) gene addition, exon skipping, stop codon read-through and gene repair in muscle tissue – and the specific challenges of genetic diseases characterized by progressive muscle degeneration
c) time-limited protein/enzyme replacement to treat genetic developmental conditions of the musculoskeletal system or the skin
d) current clinical trials and horizons of gene, cell and protein replacement therapy of genetic muscle, bone and skin disorders