Therapeutic NOTCH3 cysteine correction in CADASIL using exon skipping:in vitroproof of concept

Antisense oligonucleotides in therapy for neurodegenerative disorders

(2015) Melvin M. Evers et al.   ADVANCED DRUG DELIVERY REVIEWS

Spinal Muscular Atrophy: Therapeutic Strategies

(2014) Diana Castro et al.   Current Treatment Options in Neurology

Interpretation ofNOTCH3mutations in the diagnosis of CADASIL

(2014) Julie W Rutten et al.   EXPERT REVIEW OF MOLECULAR DIAGNOSTICS

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

(2014) Thomas Voit et al.   LANCET NEUROLOGY

Abnormal recruitment of extracellular matrix proteins by excess Notch3ECD: a new pathomechanism in CADASIL

(2013) Marie Monet-Leprêtre et al.   BRAIN

Mipomersen Sodium: First Global Approval

(2013) Philip Hair et al.   DRUGS

Hypomorphic NOTCH3 Alleles Do Not Cause CADASIL in Humans

(2013) Julie W. Rutten et al.   HUMAN MUTATION

Co-aggregate formation of CADASIL-mutant NOTCH3: a single-particle analysis

(2011) Marco Duering et al.   HUMAN MOLECULAR GENETICS

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

CADASIL

(2009) Hugues Chabriat et al.   LANCET NEUROLOGY

Evolutionary Scenarios of Notch Proteins

(2009) A. Theodosiou et al.   MOLECULAR BIOLOGY AND EVOLUTION

Amplification efficiency: linking baseline and bias in the analysis of quantitative PCR data

(2009) J. M. Ruijter et al.   NUCLEIC ACIDS RESEARCH