Y. Zhou, C. Zhang, W. Liang: Development of RNAi technology for targeted therapy–a track of siRNA based agents to RNAi therapeutics. In: Journal of controlled release : official journal of the Controlled Release Society. Band 193, November 2014, S. 270–281, doi:10.1016/j.jconrel.2014.04.044, PMID 24816071.
R. Titze-de-Almeida, C. David, S. S. Titze-de-Almeida: The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market. In: Pharmaceutical research. Band 34, Nummer 7, Juli 2017, S. 1339–1363, doi:10.1007/s11095-017-2134-2, PMID 28389707.
M. L. Bobbin, J. J. Rossi: RNA Interference (RNAi)-Based Therapeutics: Delivering on the Promise? In: Annual review of pharmacology and toxicology. Band 56, 2016, S. 103–122, doi:10.1146/annurev-pharmtox-010715-103633, PMID 26738473.
H. Ledford: Drug giants turn their backs on RNA interference. In: Nature. Band 468, Nummer 7323, November 2010, S. 487, doi:10.1038/468487a, PMID 21107398.
David J. Kuter, Herbert L. Bonkovsky, Susana Monroy, Gayle Ross, Encarna Guillén-Navarro, Maria Domenica Cappellini, Anna-Elisabeth Minder, Ole Hother-Nielsen, Paolo Ventura, Gang Jia, Marianne T. Sweetser, Manish Thapar: Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trial. In: Journal of Hepatology. Band79, Nr.5, November 2023, ISSN0168-8278, S.1150–1158, doi:10.1016/j.jhep.2023.06.013.
Sander F. Garrelfs, Yaacov Frishberg, Sally A. Hulton, Michael J. Koren, William D. O’Riordan, Pierre Cochat, Georges Deschênes, Hadas Shasha-Lavsky, Jeffrey M. Saland, William G. van’t Hoff, Daniel G. Fuster, Daniella Magen, Shabbir H. Moochhala, Gesa Schalk, Eva Simkova, Jaap W. Groothoff, David J. Sas, Kristin A. Meliambro, Jiandong Lu, Marianne T. Sweetser, Pushkal P. Garg, Akshay K. Vaishnaw, John M. Gansner, Tracy L. McGregor, John C. Lieske: Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. In: New England Journal of Medicine. Band384, Nr.13, April 2021, ISSN0028-4793, S.1216–1226, doi:10.1056/NEJMoa2021712 (nejm.org [abgerufen am 15. Juli 2024]).
David Adams, Ivailo L. Tournev, Mark S. Taylor, Teresa Coelho, Violaine Planté-Bordeneuve, John L. Berk, Alejandra González-Duarte, Julian D. Gillmore, Soon-Chai Low, Yoshiki Sekijima, Laura Obici, Chongshu Chen, Prajakta Badri, Seth M. Arum, John Vest, Michael Polydefkis, The HELIOS-A Collaborators: Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. In: Amyloid. Band30, Nr.1, 2. Januar 2023, ISSN1350-6129, S.18–26, doi:10.1080/13506129.2022.2091985 (tandfonline.com [abgerufen am 15. Juli 2024]).
Akshay S. Desai, David J. Webb, Jorg Taubel, Sarah Casey, Yansong Cheng, Gabriel J. Robbie, Don Foster, Stephen A. Huang, Sean Rhyee, Marianne T. Sweetser, George L. Bakris: Zilebesiran, an RNA Interference Therapeutic Agent for Hypertension. In: New England Journal of Medicine. Band389, Nr.3, 20. Juli 2023, ISSN0028-4793, S.228–238, doi:10.1056/NEJMoa2208391 (nejm.org [abgerufen am 15. Juli 2024]).
europa.eu
ema.europa.eu
Givlaari. European Medicines Agency, abgerufen am 15. Juli 2024.
Oxlumo. European Medicines Agency, abgerufen am 15. Juli 2024.
Sander F. Garrelfs, Yaacov Frishberg, Sally A. Hulton, Michael J. Koren, William D. O’Riordan, Pierre Cochat, Georges Deschênes, Hadas Shasha-Lavsky, Jeffrey M. Saland, William G. van’t Hoff, Daniel G. Fuster, Daniella Magen, Shabbir H. Moochhala, Gesa Schalk, Eva Simkova, Jaap W. Groothoff, David J. Sas, Kristin A. Meliambro, Jiandong Lu, Marianne T. Sweetser, Pushkal P. Garg, Akshay K. Vaishnaw, John M. Gansner, Tracy L. McGregor, John C. Lieske: Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. In: New England Journal of Medicine. Band384, Nr.13, April 2021, ISSN0028-4793, S.1216–1226, doi:10.1056/NEJMoa2021712 (nejm.org [abgerufen am 15. Juli 2024]).
Akshay S. Desai, David J. Webb, Jorg Taubel, Sarah Casey, Yansong Cheng, Gabriel J. Robbie, Don Foster, Stephen A. Huang, Sean Rhyee, Marianne T. Sweetser, George L. Bakris: Zilebesiran, an RNA Interference Therapeutic Agent for Hypertension. In: New England Journal of Medicine. Band389, Nr.3, 20. Juli 2023, ISSN0028-4793, S.228–238, doi:10.1056/NEJMoa2208391 (nejm.org [abgerufen am 15. Juli 2024]).
nih.gov
ncbi.nlm.nih.gov
Y. Zhou, C. Zhang, W. Liang: Development of RNAi technology for targeted therapy–a track of siRNA based agents to RNAi therapeutics. In: Journal of controlled release : official journal of the Controlled Release Society. Band 193, November 2014, S. 270–281, doi:10.1016/j.jconrel.2014.04.044, PMID 24816071.
R. Titze-de-Almeida, C. David, S. S. Titze-de-Almeida: The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market. In: Pharmaceutical research. Band 34, Nummer 7, Juli 2017, S. 1339–1363, doi:10.1007/s11095-017-2134-2, PMID 28389707.
M. L. Bobbin, J. J. Rossi: RNA Interference (RNAi)-Based Therapeutics: Delivering on the Promise? In: Annual review of pharmacology and toxicology. Band 56, 2016, S. 103–122, doi:10.1146/annurev-pharmtox-010715-103633, PMID 26738473.
H. Ledford: Drug giants turn their backs on RNA interference. In: Nature. Band 468, Nummer 7323, November 2010, S. 487, doi:10.1038/468487a, PMID 21107398.
David Adams, Ivailo L. Tournev, Mark S. Taylor, Teresa Coelho, Violaine Planté-Bordeneuve, John L. Berk, Alejandra González-Duarte, Julian D. Gillmore, Soon-Chai Low, Yoshiki Sekijima, Laura Obici, Chongshu Chen, Prajakta Badri, Seth M. Arum, John Vest, Michael Polydefkis, The HELIOS-A Collaborators: Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. In: Amyloid. Band30, Nr.1, 2. Januar 2023, ISSN1350-6129, S.18–26, doi:10.1080/13506129.2022.2091985 (tandfonline.com [abgerufen am 15. Juli 2024]).
David J. Kuter, Herbert L. Bonkovsky, Susana Monroy, Gayle Ross, Encarna Guillén-Navarro, Maria Domenica Cappellini, Anna-Elisabeth Minder, Ole Hother-Nielsen, Paolo Ventura, Gang Jia, Marianne T. Sweetser, Manish Thapar: Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trial. In: Journal of Hepatology. Band79, Nr.5, November 2023, ISSN0168-8278, S.1150–1158, doi:10.1016/j.jhep.2023.06.013.
Sander F. Garrelfs, Yaacov Frishberg, Sally A. Hulton, Michael J. Koren, William D. O’Riordan, Pierre Cochat, Georges Deschênes, Hadas Shasha-Lavsky, Jeffrey M. Saland, William G. van’t Hoff, Daniel G. Fuster, Daniella Magen, Shabbir H. Moochhala, Gesa Schalk, Eva Simkova, Jaap W. Groothoff, David J. Sas, Kristin A. Meliambro, Jiandong Lu, Marianne T. Sweetser, Pushkal P. Garg, Akshay K. Vaishnaw, John M. Gansner, Tracy L. McGregor, John C. Lieske: Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. In: New England Journal of Medicine. Band384, Nr.13, April 2021, ISSN0028-4793, S.1216–1226, doi:10.1056/NEJMoa2021712 (nejm.org [abgerufen am 15. Juli 2024]).
David Adams, Ivailo L. Tournev, Mark S. Taylor, Teresa Coelho, Violaine Planté-Bordeneuve, John L. Berk, Alejandra González-Duarte, Julian D. Gillmore, Soon-Chai Low, Yoshiki Sekijima, Laura Obici, Chongshu Chen, Prajakta Badri, Seth M. Arum, John Vest, Michael Polydefkis, The HELIOS-A Collaborators: Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. In: Amyloid. Band30, Nr.1, 2. Januar 2023, ISSN1350-6129, S.18–26, doi:10.1080/13506129.2022.2091985 (tandfonline.com [abgerufen am 15. Juli 2024]).
Akshay S. Desai, David J. Webb, Jorg Taubel, Sarah Casey, Yansong Cheng, Gabriel J. Robbie, Don Foster, Stephen A. Huang, Sean Rhyee, Marianne T. Sweetser, George L. Bakris: Zilebesiran, an RNA Interference Therapeutic Agent for Hypertension. In: New England Journal of Medicine. Band389, Nr.3, 20. Juli 2023, ISSN0028-4793, S.228–238, doi:10.1056/NEJMoa2208391 (nejm.org [abgerufen am 15. Juli 2024]).