Annemieke Aartsma-Rus
Annemieke Aartsma-Rus
Hoogleraar translationele genetica, Leids Universitair Medisch Centrum
Verified email at lumc.nl - Homepage
TitleCited byYear
Local dystrophin restoration with antisense oligonucleotide PRO051
JC Van Deutekom, AA Janson, IB Ginjaar, WS Frankhuizen, ...
New England Journal of Medicine 357 (26), 2677-2686, 2007
9232007
Systemic administration of PRO051 in Duchenne's muscular dystrophy
NM Goemans, M Tulinius, JT van den Akker, BE Burm, PF Ekhart, ...
New England Journal of Medicine 364 (16), 1513-1522, 2011
7582011
Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading‐frame rule
A Aartsma‐Rus, JCT Van Deutekom, IF Fokkema, GJB Van Ommen, ...
Muscle & Nerve: Official Journal of the American Association of …, 2006
5002006
Theoretic applicability of antisense‐mediated exon skipping for Duchenne muscular dystrophy mutations
A Aartsma‐Rus, I Fokkema, J Verschuuren, I Ginjaar, J Van Deutekom, ...
Human mutation 30 (3), 293-299, 2009
4622009
Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2
RJLF Lemmers, R Tawil, LM Petek, J Balog, GJ Block, GWE Santen, ...
Nature genetics 44 (12), 1370, 2012
3702012
Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients
A Aartsma-Rus, AAM Janson, WE Kaman, M Bremmer-Bout, ...
Human Molecular Genetics 12 (8), 907-914, 2003
3122003
Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense
A Aartsma-Rus, AAM Janson, WE Kaman, M Bremmer-Bout, ...
The American Journal of Human Genetics 74 (1), 83-92, 2004
2892004
Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy
A Aartsma-Rus, M Bremmer-Bout, AAM Janson, JT den Dunnen, ...
Neuromuscular Disorders 12, S71-S77, 2002
2602002
Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications
A Aartsma-Rus, GJB Van Ommen
Rna 13 (10), 1609-1624, 2007
2522007
The TREAT‐NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations
CL Bladen, D Salgado, S Monges, ME Foncuberta, K Kekou, K Kosma, ...
Human mutation 36 (4), 395-402, 2015
2262015
In vivo comparison of 2′‐O‐methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping
HA Heemskerk, CL de Winter, SJ de Kimpe, P van Kuik‐Romeijn, ...
The Journal of Gene Medicine: A cross‐disciplinary journal for research on …, 2009
2242009
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD
C Pichavant, A Aartsma-Rus, PR Clemens, KE Davies, G Dickson, ...
Molecular Therapy 19 (5), 830-840, 2011
1882011
Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle
V Arechavala-Gomeza, IR Graham, LJ Popplewell, AM Adams, ...
Human gene therapy 18 (9), 798-810, 2007
1812007
Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms
A Aartsma-Rus, L Van Vliet, M Hirschi, AAM Janson, H Heemskerk, ...
Molecular Therapy 17 (3), 548-553, 2009
1702009
Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites
A Aartsma-Rus, CL De winter, AAM Janson, WE Kaman, GB Van ommen, ...
Oligonucleotides 15 (4), 284-197, 2005
1612005
Targeted exon skipping in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotides
M Bremmer-Bout, A Aartsma-Rus, EJ De Meijer, WE Kaman, AAM Janson, ...
Molecular Therapy 10 (2), 232-240, 2004
1552004
FDA approves eteplirsen for Duchenne muscular dystrophy: the next chapter in the eteplirsen saga
A Aartsma-Rus, AM Krieg
Nucleic acid therapeutics 27 (1), 1-3, 2017
1462017
The pathogenesis and therapy of muscular dystrophies
S Guiraud, A Aartsma-Rus, NM Vieira, KE Davies, GJB van Ommen, ...
Annual review of genomics and human genetics 16, 281-308, 2015
1352015
Preclinical PK and PD studies on 2′-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model
H Heemskerk, C De Winter, P Van Kuik, N Heuvelmans, P Sabatelli, ...
Molecular Therapy 18 (6), 1210-1217, 2010
1342010
Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells
A Aartsma-Rus, WE Kaman, M Bremmer-Bout, AAM Janson, ...
Gene therapy 11 (18), 1391, 2004
1332004
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