Results
author = "Mulders, Susan A. M."
1
Found 2 documents, displaying page 1 of 1
Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide
Author(s) :
Evers, Melvin M.
,
Pepers, Barry A.
,
van Deutekom, Judith C. T.
,
Mulders, Susan A. M.
,
den Dunnen, Johan T.
,
Aartsma-Rus, Annemieke
Description :
To date there are 9 known diseases caused by an expanded polyglutamine repeat, with the most prevalent being Huntington's disease. Huntington's disease is a progressive autosomal dominant neurodegenerative disorder for which currently no therapy is available. It is caused by a CAG repeat expansion i...
Repository :
Europe PubMed Central
Language(s) :
English
Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy
Author(s) :
Mulders, Susan A. M.
,
van den Broek, Walther J. A. A.
,
Wheeler, Thurman M.
,
Croes, Huib J. E.
,
van Kuik-Romeijn, Petra
Description :
Myotonic dystrophy type 1 (DM1) is caused by toxicity of an expanded, noncoding (CUG)n tract in DM protein kinase (DMPK) transcripts. According to current evidence the long (CUG)n segment is involved in entrapment of muscleblind (Mbnl) proteins in ribonuclear aggregates and stabilized expression of ...
Repository :
Europe PubMed Central
Language(s) :
English
1
Found 2 documents, displaying page 1 of 1