Duchenne muscular dystrophy: current cell therapies

D Sienkiewicz, W Kulak… - Therapeutic …, 2015 - journals.sagepub.com
Duchenne muscular dystrophy is a genetically determined X-linked disease and the most
common, progressive pediatric muscle disorder. For decades, research has been conducted …

[图书][B] Duchenne muscular dystrophy: advances in therapeutics

JS Chamberlain, TA Rando - 2006 - books.google.com
Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of
childhood for which there is no cure. This authoritative guide provides a clear overview of …

Cell therapy strategies on Duchenne muscular dystrophy: A systematic review of clinical applications

A Akat, E Karaöz - Stem Cell Reviews and Reports, 2024 - Springer
Abstract Duchenne Muscular Dystrophy (DMD) is an inherited genetic disorder
characterized by progressive degeneration of muscle tissue, leading to functional disability …

New therapies for Duchenne muscular dystrophy: challenges, prospects and clinical trials

G Cossu, M Sampaolesi - Trends in molecular medicine, 2007 - cell.com
Muscular dystrophies primarily affect skeletal muscle. Mutations in a large number of genes,
mainly encoding cytoskeletal proteins, cause different forms of dystrophy that compromise …

Cell therapy in Duchenne muscular dystrophy treatment: clinical trials overview

A Bajek, D Porowinska, T Kloskowski… - Critical Reviews™ in …, 2015 - dl.begellhouse.com
Duchenne muscular dystrophy (DMD), the most common and most severe form of all
muscular dystrophies, leads to progressive muscle fiber necrosis, fibroblast proliferation …

Cell based therapy for Duchenne muscular dystrophy

A Farini, P Razini, S Erratico, Y Torrente… - Journal of cellular …, 2009 - Wiley Online Library
Mutations in the dystrophin gene cause an X‐linked genetic disorder: Duchenne muscular
dystrophy (DMD). Stem cell therapy is an attractive method to treat DMD because a small …

Therapeutics in duchenne muscular dystrophy

JB Strober - NeuroRx, 2006 - Springer
Duchenne muscular dystrophy (DMD) is a fatal disorder affecting approximately 1 in 3500
live born males, characterized by progressive muscle weakness. Several different strategies …

Clinical management of Duchenne muscular dystrophy: the state of the art

S Messina, GL Vita - Neurological Sciences, 2018 - Springer
Introduction Duchenne muscular dystrophy (DMD) is a devastating, progressive
neuromuscular disorder for which there is no cure. As the dystrophin gene is located on the …

Innovative therapeutic approaches for Duchenne muscular dystrophy

F Fortunato, R Rossi, MS Falzarano… - Journal of Clinical …, 2021 - mdpi.com
Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy
affecting~ 1: 5000 live male births. Following the identification of pathogenic variations in the …

Therapy of genetic disorders: novel therapies for Duchenne muscular dystrophy

JT Seto, NE Bengtsson, JS Chamberlain - Current pediatrics reports, 2014 - Springer
Duchenne muscular dystrophy is an inherited, progressive muscle-wasting disorder caused
by mutations in the dystrophin gene. An increasing variety of approaches are moving …