Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs

By J. Soutschek, Akin Akinc, B. Bramlage, K. Charissé, Rainer Constien, M. Donoghue, Sayda M. Elbashir, A. Geick, P. Hadwiger, J. Harborth and 15 more. Nature, 2004

2,198 citations recorded by the source corpus.

Subfields: Cancer Research, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/48e9d958193525d6927e435cb26c1fc033547031

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Citations in the corpus

Therapeutic EphA2 gene targeting in vivo using neutral liposomal small interfering RNA delivery. (2005, background): “have reported that siRNA conjugated with cholesterol improved delivery to multiple organs and that downregulation of ApoB was achieved in liver and jejunum (10).”
siRNA delivery systems for cancer treatment. (2009, background): “After intravenous administration in mice, conjugates of cholesterol and ApoB-specific siRNA could silence the expression of the target protein ApoB by more than 50% in the liver and jejunum, and decreased plasma levels of the ApoB protein [87].”
Delivery of siRNA Therapeutics: Barriers and Carriers (2010, methodology): “This property has been used to target the siRNA delivery to the RES organs (32, 33 ).”
Innovative approaches for cancer treatment: current perspectives and new challenges (2019, background): “Stable nucleic acid lipid particles (SNALPs) have been evaluated in non-human primates [194].”
MicroRNA: Biogenesis, Function and Role in Cancer (2010, background): “Progress is also being made in the expanding field of nanobiotechnology to develop a means to package and deliver RNA to specific sites [377-380].”
Catalytic in vivo protein knockdown by small-molecule PROTACs (2015, positive): “To confirm that this degradation is VHL dependent, we synthesized PROTAC_ ERRa_epi (2), whose stereochemistry is inverted at a key position (indicated by an asterisk in Fig.”
Strategies for silencing human disease using RNA interference (2007, methodology): “This approach was used successfully in the delivery of siRNAs into the liver and jejunum after systemic administration in mice in a study that used siRNAs targeted against apolipoprotein B ( APOB ) to modify cholesterol metabolis”
In vivo imaging of siRNA delivery and silencing in tumors (2007, background): “In fact the presence of a carrier not only preserves biological activity but also enhances stability, systemic delivery and in vivo potenc”