Thöny, Beat

Tetrahydrobiopterin: biochemistry and pathophysiology

Werner, E. R., Blau, N., & Thöny, B. (2011) · Biochemical Journal

Metabolism and Genetic DisordersFolate and B Vitamins Research

Cited by 493

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

Rothgangl, T., Dennis, M. K., Lin, P. J. C., Oka, R., Witzigmann, D., Villiger, L., Qi, W., Hruzova, M., Kissling, L., Lenggenhager, D., et al. (2021) · Nature Biotechnology

CRISPR and Genetic EngineeringRNA Interference and Gene Delivery

Cited by 378

Fusion-dependent formation of lipid nanoparticles containing macromolecular payloads

Kulkarni, J. A., Witzigmann, D., Leung, J., van der Meel, R., Zaifman, J., Darjuan, M. M., Grisch-Chan, H. M., Thöny, B., Tam, Y. Y. C., & Cullis, P. R. (2019) · Nanoscale

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 161

In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA

Villiger, L., Rothgangl, T., Witzigmann, D., Oka, R., Lin, P. J. C., Qi, W., Janjuha, S., Berk, C., Ringnalda, F., Beattie, M. B., et al. (2021) · Nature Biomedical Engineering

CRISPR and Genetic EngineeringRNA regulation and disease

Cited by 105

Enhancement of hepatic autophagy increases ureagenesis and protects against hyperammonemia

Soria, L. R., Allegri, G., Melck, D., Pastore, N., Annunziata, P., Paris, D., Polishchuk, E., Nusco, E., Thöny, B., Motta, A., et al. (2018) · Proceedings of the National Academy of Sciences

Autophagy in Disease and TherapyMetabolism and Genetic Disorders

Cited by 61