Stem Cell & Gene Therapies for Skeletal Muscle: New Era, New Opportunities

Stem Cell & Gene Therapies for Skeletal Muscle: New Era, New Opportunities

8-9 May 2018. Homerton College, Cambridge

Homerton College, Cambridge

8-9 MAY 2018

‘Stem Cell & Gene Therapies for Skeletal Muscle: New Era, New Opportunities’

Two-day intensive course for researchers, R&D professionals and clinical practitioners 

The musculoskeletal system provides shape, support, stability, and movement to the body. It is made up of the bones of the skeleton, muscles, cartilage, tendons, ligaments, joints, and other connective tissue that give us form and function. Skeletal muscle is the contractile element of this system and by itself, accounts for about 30-50% of our body weight(1). In addition to movement, muscle contraction also fulfils some other important functions in the body, such as posture, joint stability, and heat production(2). Skeletal muscle has outstanding adaptive and regenerative capacity throughout life. This capacity for growth, repair and regeneration is endowed by its resident population of stem cells, which are called ‘satellite cells’(3,4).

Given the many important functions of muscle it is perhaps not surprising that a loss of muscle mass or an impaired regenerative capacity, can lead to weakness and disability, decreased independence, reduced quality of life and increased mortality (2). Loss of muscle volume and function are typical of conditions like such as in accompanies ageing (a process called sarcopenia)(1,2,5), prolonged inactivity, denervation, acute or chronic illness (cachexia) (6), or inherited or acquired muscle diseases (Skeletal muscle myopathies), or a reduction in gravity (space flight).

Skeletal muscle myopathies including congentital myothpathies, muscular dystrophies and inflammatory myopathies are neuromuscular disorders in which the primary symptom is muscle weakness due to dysfunction of the muscle fibre, often accompanied by a loss of functional muscle mass. The prognosis for individuals with a myopathy varies but in some cases the disorder may be progressive, severely disabling, life threatening, or fatal.

Duchenne muscular dystrophy (DMD) is the most common X-linked genetic disorder in humans; it affects one in 3,500 males. Most boys with DMD manifest symptoms within the first years of life (1). The majority of DMD patients die prematurely as a result of respiratory or cardiovascular failure in their twenties. The cause of DMD is known to be mutations in the dystrophin gene, yet still a cure has remained elusive(1).

Treatments for the myopathies vary depending on the disease, condition or specific cause. For some disorders of the muscle, especially those with genetic underpinnings, only supportive or symptomatic treatments are currently available. However, a growing understanding of the stem and progenitor populations in skeletal muscle, along with the advent of new genetic engineering technologies, including CRISPR-Cas9 (and variations of this), and increasingly advanced pharmacological therapeutics, we are now entering a era when better treatments are appearing on the horizon (7-9).





In this two-day intensive course by CamBioScience you will study the importance of the musculoskeletal system for normal function and delve into the stem cell populations, which give it its remarkable regenerative capacity. You will learn about the pathophysiological mechanism of muscle diseases as well as the inevitable muscle wasting that accompanies human ageing. Research on the cellular and genetic solutions for preventing, ameliorating and potentially curing muscle wasting and degeneration will be discussed with input from both scientific and medical specialists aiming to make get new and effective treatments approved in the clinics. Finally you will sit an exam and receive certification reflecting you commitment to maintaining a world-class knowledge and skill base in skeletal muscle disease and therapies.

On this course you will:

  • Gain insights into the development of the musculoskeletal system.
  • Understand the cellular players involved in muscle regeneration and how these might be leveraged for therapeutic gain.
  • Learn about key molecular pathways involved in skeletal muscle growth, atrophy, regeneration and denegation.
  • Better understand the aetiology of diseases muscle disease, especially those with genetic underpinnings (e.g. DMD).
  • Engage with the latest research in skeletal muscle biology with leading researchers in the field.
  • Acquire an understanding of the newest tools and technologies available for investigating the molecular genetics of muscle stem and progenitor cells in health and disease.
  • iPS cells for modelling muscular dystrophies.
  • Gain exclusive information of advances in drug discovery for skeletal muscle wasting and disease.
  • Review the state-of-play and upcoming gene and cell based therapeutic opportunities for muscle wasting diseases.
  • Have a forum to present and promote your work to an international audience.

Who is this course aimed at?

  • Research students
  • Postdoctoral researchers
  • R&D professionals from industry
  • Junior/Senior Research Group leaders
  • Medical Doctors/Orthapedics surgeons


  • Participants successfully completing the course exam will receive a certificate of completion.
  • The course will be CPD accredited.







Professor Peter Zammit, King’s College London

Professor Jennifer Morgan, University College London

Professor Steve Harridge, King’s College London

Dr Francesco Saverio Tedesco, University College London

> more about the team


(1) Skeletal Muscle Degenerative Diseases and Strategies for Therapeutic Muscle Repair. Tabebordbar et al. (2013). Annual Reviews in Pathology. 8, 441-75.

(2) Harridge, S. D. R. (2007). Plasticity of human skeletal muscle: gene expression to in vivo function. Experimental Physiology. 92, 5, p. 783 – 797

(3) Zammit P et al. The skeletal muscle satellite cell: the stem cell that came in from the cold (2006). J Histochem Cytochem. 54(11):1177-91.

(4) PAX7 target genes are globally repressed in facioscapulohumeral muscular dystrophy skeletal muscle. Banerji, et al. (2017). Nature Communications. 8,1, 2152.

(5) Brack & Muñoz-Cánoves. The ins and outs of muscle stem cell aging. Skeletal Muscle, 20166: 1

(6) Puthucheary ZA (2013). Acute skeletal muscle wasting in critical illness. JAMA. 310(15):1591-600.

(7) Francesco Saverio Tedesco et al. (2010).  Repairing skeletal muscle: regenerative potential of skeletal muscle stem cells. J. Clin. Invest. 120, 11–19

(8) O’Neill, H. C., Pini, V., Muntoni, F., & Morgan, J. (2017). Genome Editing and Muscle Stem Cells as a Therapeutic Tool for Muscular Dystrophies. Current Stem Cell Reports.

(9) Francesco Saverio Tedesco et al. (2011). Stem Cell–Mediated Transfer of a Human Artificial Chromosome Ameliorates Muscular Dystrophy. Science Translational Medicine. 3 (96), 96ra78.

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