[HTML][HTML] Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy
D Duan - Molecular Therapy, 2018 - cell.com
Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …
AAV-based gene therapies for the muscular dystrophies
JM Crudele, JS Chamberlain - Human molecular genetics, 2019 - academic.oup.com
Muscular dystrophy (MD) is a group of progressive genetic diseases affecting the
musculature that are characterized by inflammatory infiltrates, necrosis and connective …
musculature that are characterized by inflammatory infiltrates, necrosis and connective …
Gene therapies in canine models for Duchenne muscular dystrophy
PP Nghiem, JN Kornegay - Human Genetics, 2019 - Springer
Therapies for Duchenne muscular dystrophy (DMD) must first be tested in animal models to
determine proof-of-concept, efficacy, and importantly, safety. The murine and canine models …
determine proof-of-concept, efficacy, and importantly, safety. The murine and canine models …
[HTML][HTML] The dog model in the spotlight: legacy of a trustful cooperation
Dogs have long been used as a biomedical model system and in particular as a preclinical
proof of concept for innovative therapies before translation to humans. A recent example of …
proof of concept for innovative therapies before translation to humans. A recent example of …
Gene Replacement Therapy for Duchenne Muscular Dystrophy
K Hollinger, JM Crudele, JS Chamberlain - Muscle Gene Therapy, 2019 - Springer
Duchenne muscular dystrophy (DMD) is a fatal, X-linked disease caused by mutations in the
massive dystrophin gene that lead to extremely low or non-detectable levels of dystrophin …
massive dystrophin gene that lead to extremely low or non-detectable levels of dystrophin …
Muscular Dystrophy
K Hollinger, JM Crudele… - Muscle Gene Therapy, 2019 - books.google.com
Duchenne muscular dystrophy (DMD) is a fatal, X-linked disease caused by mutations in the
massive dystrophin gene that lead to extremely low or non-detectable levels of dystrophin …
massive dystrophin gene that lead to extremely low or non-detectable levels of dystrophin …