Precision medicine using whole genome sequencing identifies a novel dystrophin (DMD) variant for X‐linked muscular dystrophy in a cat
GD Shelton, F Tucciarone, LT Guo… - Journal of Veterinary …, 2024 - Wiley Online Library
Abstract Background Muscular dystrophies (MDs) are a large, heterogeneous group of
degenerative muscle diseases. X‐linked dystrophin‐deficient MD in cats is the first …
degenerative muscle diseases. X‐linked dystrophin‐deficient MD in cats is the first …
[HTML][HTML] CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments
In recent years, clustered regularly interspaced short palindromic repeats (CRISPRs) and
CRISPR-associated (Cas) protein have emerged as a revolutionary gene editing tool to treat …
CRISPR-associated (Cas) protein have emerged as a revolutionary gene editing tool to treat …
[HTML][HTML] Is Cardiac Transplantation Still a Contraindication in Patients with Muscular Dystrophy-Related End-Stage Dilated Cardiomyopathy? A Systematic Review
L Politano - International Journal of Molecular Sciences, 2024 - mdpi.com
Inherited muscular diseases (MDs) are genetic degenerative disorders typically caused by
mutations in a single gene that affect striated muscle and result in progressive weakness …
mutations in a single gene that affect striated muscle and result in progressive weakness …
[HTML][HTML] Mechanisms of Chimeric Cell Therapy in Duchenne Muscular Dystrophy
M Siemionow, A Ziemiecka, K Bożyk, K Siemionow - Biomedicines, 2024 - mdpi.com
Despite scientific efforts, there is no cure for Duchenne muscular dystrophy (DMD), a lethal,
progressive, X-linked genetic disorder caused by mutations in the dystrophin gene. DMD …
progressive, X-linked genetic disorder caused by mutations in the dystrophin gene. DMD …
[HTML][HTML] Gene therapy delivered micro-dystrophins co-localize with transgenic utrophin in dystrophic skeletal muscle fibers
S Krishna, AB Piepho, DM Lake, LR Cumby… - Neuromuscular …, 2024 - Elsevier
Duchenne muscular dystrophy (DMD) is a devastating muscle disease caused by the
absence of functional dystrophin. There are multiple ongoing clinical trials for DMD that are …
absence of functional dystrophin. There are multiple ongoing clinical trials for DMD that are …