scholarly journals Adenine base editing of the DUX4 polyadenylation signal for targeted genetic therapy in Facioscapulohumeral muscular dystrophy

2021 ◽  
Author(s):  
Darina Šikrová ◽  
Vlad A. Cadar ◽  
Yavuz Ariyurek ◽  
Jeroen F.J. Laros ◽  
Judit Balog ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Facioscapulohumeral Muscular Dystrophy ◽  
Polyadenylation Signal ◽  
Genetic Therapy ◽  
Base Editing ◽  
Adenine Base

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

2018 ◽  
Vol 36 (6) ◽  
pp. 536-539 ◽  
Author(s):  
Seuk-Min Ryu ◽  
Taeyoung Koo ◽  
Kyoungmi Kim ◽  
Kayeong Lim ◽  
Gayoung Baek ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Adult Mouse ◽  
Mouse Embryos ◽  
Base Editing ◽  
Adenine Base

scholarly journals Facioscapulohumeral muscular dystrophy – Reproductive counseling, pregnancy and delivery in a complex multigenetic disease

2021 ◽  
Author(s):  
Sanne C. C. Vincenten ◽  
Nienke Stoep ◽  
Aimée D. C. Paulussen ◽  
Karlien Mul ◽  
Umesh A. Badrising ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Facioscapulohumeral Muscular Dystrophy ◽  
Pregnancy And Delivery ◽  
Reproductive Counseling

Magnetic resonance imaging in a large cohort of facioscapulohumeral muscular dystrophy patients: Pattern refinement and implications for clinical trials

2016 ◽  
Vol 79 (5) ◽  
pp. 854-864 ◽  
Author(s):  
Giorgio Tasca ◽  
Mauro Monforte ◽  
Pierfrancesco Ottaviani ◽  
Marco Pelliccioni ◽  
Roberto Frusciante ◽  
...  
Keyword(s):  
Magnetic Resonance Imaging ◽  
Clinical Trials ◽  
Magnetic Resonance ◽  
Muscular Dystrophy ◽  
Facioscapulohumeral Muscular Dystrophy ◽  
Large Cohort ◽  
Resonance Imaging

Disease modifying factors in facioscapulohumeral muscular dystrophy: Protocol of the FSHD-FOCUS study

2015 ◽  
Vol 25 ◽  
pp. S214-S215
Author(s):  
K. Mul ◽  
G. Horlings ◽  
R. Lemmers ◽  
N. Voermans ◽  
S. van der Maarel ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Facioscapulohumeral Muscular Dystrophy ◽  
Modifying Factors ◽  
Disease Modifying
Sign in / Sign up

Export Citation Format

Share Document