INNOVATIVE SPLICE SWITCHING OLIGONUCLEOTIDE THERAPY FOR NIEMANN-PICK TYPE C1 DISEASE

• Maria Martinez De Lagran,
• Yu-Han Huang,
• Ondrej Kostov,
• Haiyan Zhou,
• Paul Gissen,
• Marvin Caruthers,
• Timothy Yu,
• Daniel Ginberg,
• Mara Dierssen

Affiliations

Maria Martinez De Lagran

Centro de Regulacion Genomica, Systems And Synthetic Biology, Barcelona, Spain; Centro de Investigacion Biomedica en Red, Ciberer, Madrid, Spain

Yu-Han Huang

Harvard Medical School, Division Of Genetics And Genomics, Boston, United States of America

Ondrej Kostov

University of Colorado, Department Of Biochemistry, Boulder, United States of America

Haiyan Zhou

Great Ormond Street Institute of Child Health, University College London, Genetics And Genomic Medicine Department, London, United Kingdom

Paul Gissen

Great Ormond Street Institute of Child Health, University College London, Genetics And Genomic Medicine Department, London, United Kingdom

Marvin Caruthers

University of Colorado, Department Of Biochemistry, Boulder, United States of America

Timothy Yu

Harvard Medical School, Division Of Genetics And Genomics, Boston, United States of America

Daniel Ginberg

Faculty of Biology, Universitat de Barcelona, Department Of Genetics, Microbiology And Statistic, Barcelona, Spain

Mara Dierssen

Centro de Regulacion Genomica, Systems And Synthetic Biology, Barcelona, Spain; Centro de Investigacion Biomedica en Red, Ciberer, Madrid, Spain