Safe and Effective Cynomolgus Monkey GLP-Tox Study with Repetitive Intrathecal Application of a TGFBR2 Targeting LNA-Gapmer Antisense Oligonucleotide as Treatment Candidate for Neurodegenerative Disorders.
Peters, S; Wirkert, E; Kuespert, S; Heydn, R; Johannesen, S; Friedrich, A; Mailander, S; Korte, S; Mecklenburg, L; Aigner, L; Bruun, TH; Bogdahn, U
PHARMACEUTICS. 2022; 14(1):
Full papers/articles (Journal)

Targeting TGF-ss in the Central Nervous System: Assessment of Cynomolgus Monkey-Toxicity and Pharmacokinetics for an LNA-Antisense Oligonucleotide
Peters, S; Wirkert, E; Kuespert, S; Heydn, R; Korte, S; Mecklenburg, L; Aigner, L; Johannesen, S; Bruun, TH; Bogdahn, U
APPL SCI-BASEL. 2022; 12(3): 973
Full papers/articles (Journal)

Personalized Development of Antisense Oligonucleotides for Exon Skipping Restores Type XVII Collagen Expression in Junctional Epidermolysis Bullosa.
Ablinger, M; Lettner, T; Friedl, N; Potocki, H; Palmetzhofer, T; Koller, U; Illmer, J; Liemberger, B; Hainzl, S; Klausegger, A; Reisenberger, M; Lambert, J; Van Gele, M; Desmet, E; Van Maelsaeke, E; Wimmer, M; Zauner, R; Bauer, JW; Wally, V;
Int J Mol Sci. 2021; 22(7):
Full papers/articles (Journal)

QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study.
Bornert, O; Hogervorst, M; Nauroy, P; Bischof, J; Swildens, J; Athanasiou, I; Tufa, SF; Keene, DR; Kiritsi, D; Hainzl, S; Murauer, EM; Marinkovich, MP; Platenburg, G; Hausser, I; Wally, V; Ritsema, T; Koller, U; Haisma, EM; Nyström, A;
J Invest Dermatol. 2021; 141(4):883-893.e6
Full papers/articles (Journal)

Reconditioning the Neurogenic Niche of Adult Non-human Primates by Antisense Oligonucleotide-Mediated Attenuation of TGFβ Signaling.
Peters, S; Kuespert, S; Wirkert, E; Heydn, R; Jurek, B; Johannesen, S; Hsam, O; Korte, S; Ludwig, FT; Mecklenburg, L; Mrowetz, H; Altendorfer, B; Poupardin, R; Petri, S; Thal, DR; Hermann, A; Weishaupt, JH; Weis, J; Aksoylu, IS; Lewandowski, SA; Aigner, L; Bruun, TH; Bogdahn, U
Neurotherapeutics. 2021; 18(3):1963-1979
Full papers/articles (Journal)

Clinical Perspectives of Gene-Targeted Therapies for Epidermolysis Bullosa.
Welponer, T; Prodinger, C; Pinon-Hofbauer, J; Hintersteininger, A; Breitenbach-Koller, H; Bauer, JW; Laimer, M;
Dermatol Ther (Heidelb). 2021; 11(4): 1175-1197.
Reviews

Novel regulators of PrP
Colini Baldeschi, A; Vanni, S; Zattoni, M; Legname, G;
Expert Opin Ther Targets. 2020; 24(8):759-776
Reviews

Antisense Oligonucleotide in LNA-Gapmer Design Targeting TGFBR2-A Key Single Gene Target for Safe and Effective Inhibition of TGFβ Signaling.
Kuespert, S; Heydn, R; Peters, S; Wirkert, E; Meyer, AL; Sieborger, M; Johannesen, S; Aigner, L; Bogdahn, U; Bruun, TH
INT J MOL SCI. 2020; 21(6):
Full papers/articles (Journal)

"Transcriptomics": molecular diagnosis of inborn errors of metabolism via RNA-sequencing.
Kremer, LS; Wortmann, SB; Prokisch, H
J INHERIT METAB DIS. 2018; 41(3): 525-532.
Full papers/articles (Journal)

RNA Trans-Splicing Modulation via Antisense Molecule Interference.
Liemberger, B; Piñón Hofbauer, J; Wally, V; Arzt, C; Hainzl, S; Kocher, T; Murauer, EM; Bauer, JW; Reichelt, J; Koller, U;
Int J Mol Sci. 2018; 19(3):
Full papers/articles (Journal)

RNA-based therapies for genodermatoses.
Bornert, O; Peking, P; Bremer, J; Koller, U; van den Akker, PC; Aartsma-Rus, A; Pasmooij, AM; Murauer, EM; Nyström, A;
Exp Dermatol. 2017; 26(1):3-10
Reviews

Identification of Genes Controlled by the Essential YycFG Two-Component System Reveals a Role for Biofilm Modulation in Staphylococcus epidermidis.
Xu, T; Wu, Y; Lin, ZW; Bertram, R; Gotz, F; Zhang, Y; Qu, D
FRONT MICROBIOL. 2017; 8: 724
Full papers/articles (Journal)

Trans-splicing improvement by the combined application of antisense strategies.
Koller, U; Hainzl, S; Kocher, T; Hüttner, C; Klausegger, A; Gruber, C; Mayr, E; Wally, V; Bauer, JW; Murauer, EM;
Int J Mol Sci. 2015; 16(1):117-191
Full papers/articles (Journal)

Polymorphisms in the apoptotic pathway gene BCL-2 and survival in small cell lung cancer.
Knoefel, LF; Werle-Schneider, G; Dally, H; Müller, PJ; Edler, L; Bartsch, H; Tuengerthal, S; Heussel, CP; Reinmuth, N; Thomas, M; Risch, A;
J Thorac Oncol. 2011; 6(1): 183-189.
Full papers/articles (Journal)

Inhibition of Smad7, a negative regulator of TGF-beta signaling, suppresses autoimmune encephalomyelitis.
Kleiter, I; Pedré, X; Mueller, AM; Poeschl, P; Couillard-Despres, S; Spruss, T; Bogdahn, U; Giegerich, G; Steinbrecher, A;
J Neuroimmunol. 2007; 187(1-2):61-73
Full papers/articles (Journal)

Integrative elements for Bacillus subtilis yielding tetracycline-dependent growth phenotypes.
Bertram, R; Köstner, M; Müller, J; Vazquez Ramos, J; Hillen, W;
Nucleic Acids Res. 2005; 33(18):e153
Full papers/articles (Journal)

Nucleic acid-based modulation of cardiac gene expression for the treatment of cardiac diseases. Approaches and perspectives.
Poller, W; Fechner, H; Kurreck, J; Pauschinger, M; Kühl, U; Erdmann, VA; Lamers, JM; Schultheiss, HP;
Z Kardiol. 2004; 93(3):171-193
Reviews

Fluorescence-optical measurements of chloride movements in cells using the membrane-permeable dye diH-MEQ.
Wöll, E; Gschwentner, M; Fürst, J; Hofer, S; Buemberger, G; Jungwirth, A; Frick, J; Deetjen, P; Paulmichl, M;
Pflugers Arch. 1996; 432(3): 486-493.
Full papers/articles (Journal)

Antisense oligonucleotides suppress cell-volume-induced activation of chloride channels.
Gschwentner, M; Nagl, UO; Wöll, E; Schmarda, A; Ritter, M; Paulmichl, M;
Pflugers Arch. 1995; 430(4): 464-470.
Full papers/articles (Journal)

Thyroid hormone influences conditional transcript elongation of the apolipoprotein A-I gene in rat liver.
Lin-Lee, YC; Soyal, SM; Surguchov, A; Sanders, S; Strobl, W; Patsch, W;
J Lipid Res. 1995; 36(7):1586-1594
Full papers/articles (Journal)

In situ hybridization for the detection of low copy numbers of c-abl oncogene mRNA in lymphoma cells: technical approach and comparison with results with anti-oncoprotein antibodies.
Greil, R; Fasching, B; Huber, H;
Lab Invest. 1989; 60(4): 574-582.
Full papers/articles (Journal)