Nonclinical Exon Skipping Studies with 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results

por van Putten, Maaike, Tanganyika-de Winter, Christa, Bosgra, Sieto, Aartsma-Rus, Annemieke
Publicado 2019

Assessment of the feasibility of exon 45–55 multiexon skipping for duchenne muscular dystrophy

por van Vliet, Laura, de Winter, Christa L, van Deutekom, Judith CT, van Ommen, Gert-Jan B, Aartsma-Rus, Annemieke
Publicado 2008

Long-term Exon Skipping Studies With 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models

por Tanganyika-de Winter, Christa L, Heemskerk, Hans, Karnaoukh, Tatyana G, van Putten, Maaike, de Kimpe, Sjef J, van Deutekom, Judith, Aartsma-Rus, Annemieke
Publicado 2012

Cross-sectional study into age-related pathology of mouse models for limb girdle muscular dystrophy types 2D and 2F

por Verhaart, Ingrid E. C., Putker, Kayleigh, van de Vijver, Davy, Tanganyika-de Winter, Christa L., Pasteuning-Vuhman, Svetlana, Plomp, Jaap J., Aartsma-Rus, Annemieke M., van Putten, Maaike
Publicado 2019

Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

por Jirka, Silvana M G, Tanganyika-de Winter, Christa L, Boertje-van der Meulen, Joke W, van Putten, Maaike, Hiller, Monika, Vermue, Rick, de Visser, Peter C, Aartsma-Rus, Annemieke
Publicado 2015

Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy

por Jirka, Silvana M.G., ’t Hoen, Peter A.C., Diaz Parillas, Valeriano, Tanganyika-de Winter, Christa L., Verheul, Ruurd C., Aguilera, Begona, de Visser, Peter C., Aartsma-Rus, Annemieke M.
Publicado 2018

Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy

por Verhaart, Ingrid E.C., Cappellari, Ornella, Tanganyika-de Winter, Christa L., Plomp, Jaap J., Nnorom, Sofia, Wells, Kim E., Hildyard, John C.W., Bull, David, Aartsma-Rus, Annemieke, Wells, Dominic J.
Publicado 2021

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

por Heemskerk, Hans, de Winter, Christa, van Kuik, Petra, Heuvelmans, Niki, Sabatelli, Patrizia, Rimessi, Paola, Braghetta, Paola, van Ommen, Gert-Jan B, de Kimpe, Sjef, Ferlini, Alessandra, Aartsma-Rus, Annemieke, van Deutekom, Judith CT
Publicado 2010

Peripheral blood transcriptome profiling enables monitoring disease progression in dystrophic mice and patients

por Signorelli, Mirko, Ebrahimpoor, Mitra, Veth, Olga, Hettne, Kristina, Verwey, Nisha, García‐Rodríguez, Raquel, Tanganyika‐deWinter, Christa L, Lopez Hernandez, Luz B, Escobar Cedillo, Rosa, Gómez Díaz, Benjamín, Magnusson, Olafur T, Mei, Hailiang, Tsonaka, Roula, Aartsma‐Rus, Annemieke, Spitali, Pietro
Publicado 2021

Dystrophin deficiency leads to dysfunctional glutamate clearance in iPSC derived astrocytes

por Patel, Abdulsamie M., Wierda, Keimpe, Thorrez, Lieven, van Putten, Maaike, De Smedt, Jonathan, Ribeiro, Luis, Tricot, Tine, Gajjar, Madhavsai, Duelen, Robin, Van Damme, Philip, De Waele, Liesbeth, Goemans, Nathalie, Tanganyika-de Winter, Christa, Costamagna, Domiziana, Aartsma-Rus, Annemieke, van Duyvenvoorde, Hermine, Sampaolesi, Maurilio, Buyse, Gunnar M., Verfaillie, Catherine M.
Publicado 2019