Molecular therapy - Nucleic acids, 2162-2531

Journal

Related Publications
  1. Combining Engineered U1 snRNA and Antisense Oligonucleotides to Improve the Treatment of a BBS1 Splice Site Mutation

    Breuel, S., Vorm, M., Braeuer, A. U., Owczarek-Lipska, M. & Neidhardt, J., 6-Dec-2019, In : Molecular therapy - Nucleic acids. 18, p. 123-130 8 p.

    Research output: Contribution to journalArticleAcademicpeer-review

  2. Natural Exon Skipping Sets the Stage for Exon Skipping as Therapy for Dystrophic Epidermolysis Bullosa

    Bremer, J., van der Heijden, E. H., Eichhorn, D. S., Meijer, R., Lemmink, H. H., Scheffer, H., Sinke, R. J., Jonkman, M. F., Pasmooij, A. M. G. & Van den Akker, P. C., 18-Dec-2019, In : Molecular therapy - Nucleic acids. 18, p. 465-475 11 p.

    Research output: Contribution to journalArticleAcademicpeer-review

  3. Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa: Exon Skipping as Systemic Therapy for RDEB

    Bremer, J., Bornert, O., Nyström, A., Gostynski, A., Jonkman, M. F., Aartsma-Rus, A., van den Akker, P. C. & Pasmooij, A. MG., 2016, In : Molecular therapy - Nucleic acids. 5, 9 p., e379.

    Research output: Contribution to journalArticleAcademicpeer-review

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