Therapeutic developments for Duchenne muscular dystrophy

IEC Verhaart, A Aartsma-Rus - Nature Reviews Neurology, 2019 - nature.com
Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Therapeutic approaches for Duchenne muscular dystrophy

TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Emerging therapies for Duchenne muscular dystrophy

T Markati, M Oskoui, MA Farrar, T Duong… - The Lancet …, 2022 - thelancet.com
Duchenne muscular dystrophy is an X-linked disease caused by the absence of functional
dystrophin in the muscle cells. Major advances have led to the development of gene …

Advances in stem cell research and therapeutic development

M De Luca, A Aiuti, G Cossu, M Parmar… - Nature cell …, 2019 - nature.com
Despite many reports of putative stem-cell-based treatments in genetic and degenerative
disorders or severe injuries, the number of proven stem cell therapies has remained small …

Stem cells in the treatment of disease

HM Blau, GQ Daley - New England Journal of Medicine, 2019 - Mass Medical Soc
Stem-Cell Research and Clinical Application Stem cells have been the focus of hope, hype,
and a great deal of research. But what exactly is a stem cell, and what are its uses? This …

Lancet Commission: Stem cells and regenerative medicine

G Cossu, M Birchall, T Brown, P De Coppi… - The Lancet, 2018 - thelancet.com
Executive summary In this Commission, we argue that a combination of poor quality science,
unclear funding models, unrealistic hopes, and unscrupulous private clinics threatens …

Muscle injuries and strategies for improving their repair

T Laumonier, J Menetrey - Journal of experimental orthopaedics, 2016 - Springer
Satellite cells are tissue resident muscle stem cells required for postnatal skeletal muscle
growth and repair through replacement of damaged myofibers. Muscle regeneration is …

[HTML][HTML] Regulation and phylogeny of skeletal muscle regeneration

MB Baghdadi, S Tajbakhsh - Developmental biology, 2018 - Elsevier
One of the most fascinating questions in regenerative biology is why some animals can
regenerate injured structures while others cannot. Skeletal muscle has a remarkable …

Stem cell therapy for muscular dystrophies

S Biressi, A Filareto, TA Rando - The Journal of Clinical …, 2020 - Am Soc Clin Investig
Muscular dystrophies are a heterogeneous group of genetic diseases, characterized by
progressive degeneration of skeletal and cardiac muscle. Despite the intense investigation …

An overview about the biology of skeletal muscle satellite cells

L Forcina, C Miano, L Pelosi, A Musarò - Current genomics, 2019 - ingentaconnect.com
The peculiar ability of skeletal muscle tissue to operate adaptive changes during post-natal
development and adulthood has been associated with the existence of adult somatic stem …