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Improvement of DNA vector delivery of DOTAP lipoplexes by short-chain aminolipids

Buck, Jonas ; Mueller, Dennis ; Mettal, Ute ; Ackermann, Miriam ; Grisch-Chan, Hiu Man ; Thöny, Beat ; Zumbuehl, Andreas ; Huwyler, Jörg ; Witzigmann, Dominik

In: ACS Omega, 2020, vol. 5, no. 38, p. 24724–24732

Cellular delivery of DNA vectors for the expression of therapeutic proteins is a promising approach to treat monogenic disorders or cancer. Significant efforts in a preclinical and clinical setting have been made to develop potent nonviral gene delivery systems based on lipoplexes composed of permanently cationic lipids. However, transfection efficiency and tolerability of such systems are in...

Tetrahydrobiopterin in biomedical research

Blau, Nenad ; Thöny, Beat

In: Journal of Inherited Metabolic Disease, 2009, vol. 32, no. 1, p. 1-2

Erratum to: Diagnosis of tetrahydrobiopterin deficiency using filter paper blood spots: further development of the method and 5 years experience

Opladen, Thomas ; Abu Seda, Bettina ; Rassi, Anahita ; Thöny, Beat ; Hoffmann, Georg ; Blau, Nenad

In: Journal of Inherited Metabolic Disease, 2011, vol. 34, no. 3, p. 843-843

Diagnosis of tetrahydrobiopterin deficiency using filter paper blood spots: further development of the method and 5 years experience

Opladen, Thomas ; Abu Seda, Bettina ; Rassi, Anahita ; Thöny, Beat ; Hoffmann, Georg ; Blau, Nenad

In: Journal of Inherited Metabolic Disease, 2011, vol. 34, no. 3, p. 819-826

Long-term correction of murine phenylketonuria by viral gene transfer: liver versus muscle

Thöny, Beat

In: Journal of Inherited Metabolic Disease, 2010, vol. 33, no. 6, p. 677-680

Severe mucitis after sublingual administration of tetrahydrobiopterin in a patient with tetrahydrobiopterin-responsive phenylketonuria

Opladen, Thomas ; Zurflüh, Marcel ; Kern, Ilse ; Kierat, Lucja ; Thöny, Beat ; Blau, Nenad

In: European Journal of Pediatrics, 2005, vol. 164, no. 6, p. 395-396

Isolation of 6-Pyruvoyl-tetrahydropterin Synthase cDNAs from Human Brain

Kluge, Claudia ; Leimbacher, Walter ; Heizmann, Claus W. ; Blau, Nenad ; Thöny, Beat

In: Pteridines, 1996, vol. 7, no. 3, p. 91-93

Tetrahydrobiopterin Deficiency: From Phenotype to Genotype

Blau, Nenad ; Thöny, Beat ; Heizmann, Claus W. ; Dhondt, Jean-Louis

In: Pteridines, 1993, vol. 4, no. 1, p. 1-10

Brain catecholamine depletion and motor impairment in a Th knock-in mouse with type B tyrosine hydroxylase deficiency

Korner, Germaine

In: Brain, 2015, vol. 138, no. 10, p. 2948-2963

Characterization of Human Pterin-4a-carbinolamine Dehydratase/Dimerization Cofactor of Hepatocyte Nuclear Factor-1α, and of the Cys81-mutant Involved in Hyperphenylalanincemia

Köster, Sandra ; Thöny, Beat ; Macheroux, Peter ; Curtius, Hans-Christoph ; Heizmann, Claus W. ; Ghisla, Sandro

In: Pteridines, 1995, vol. 6, no. 3, p. 123-125