Influence of Cationic Lipid Composition on Gene Silencing Properties of Lipid Nanoparticle Formulations of siRNA in Antigen-Presenting Cells
Genc Basha,Tatiana Novobrantseva,Nicole L. Rosin,Yuen Yi C. Tam,Ismail M. Hafez,Matthew K Wong,Tsukasa Sugo,Vera M. Ruda,June Qin,Boris Klebanov,Marco A. Ciufolini,Akin Akinc,Ying K. Tam,Michael J. Hope,Pieter R. Cullis +14 more
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TLDR
In vivo results showed that LNP siRNA systems containing DLinKC2-DMA are effective agents for silencing GAPDH in APCs in the spleen and peritoneal cavity following systemic administration.About:
This article is published in Molecular Therapy.The article was published on 2011-12-01 and is currently open access. It has received 198 citations till now. The article focuses on the topics: Small interfering RNA & Gene silencing.read more
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Journal ArticleDOI
Liposomal drug delivery systems: from concept to clinical applications.
TL;DR: Lipidic nanoparticles are the first nanomedicine delivery system to make the transition from concept to clinical application, and they are now an established technology platform with considerable clinical acceptance.
Journal ArticleDOI
Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
John A. Zuris,David B. Thompson,Yilai Shu,John Paul Guilinger,Jeffrey L. Bessen,Johnny Hao Hu,Morgan L. Maeder,J. Keith Joung,Zheng-Yi Chen,David R. Liu +9 more
TL;DR: It is reported that common cationic lipid nucleic acid transfection reagents can potently deliver proteins that are fused to negatively supercharged proteins, that contain natural anionic domains or that natively bind to anionic nucleic acids.
Journal ArticleDOI
Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape
Jerome Gilleron,William Querbes,Anja Zeigerer,Anna Borodovsky,Giovanni Marsico,Undine Schubert,Kevin Manygoats,Sarah Seifert,Cordula Andree,Martin Stöter,Hila Epstein-Barash,Ligang Zhang,Victor Koteliansky,Kevin Fitzgerald,Eugenio Fava,Marc Bickle,Yannis Kalaidzidis,Akin Akinc,Martin Maier,Marino Zerial +19 more
TL;DR: It is estimated that escape of siRNAs from endosomes into the cytosol occurs at low efficiency (1–2%) and only during a limited window of time when the LNPs reside in a specific compartment sharing early and late endosomal characteristics.
Journal ArticleDOI
Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling
Gaurav Sahay,William Querbes,Christopher A. Alabi,Ahmed A. Eltoukhy,Sovan Sarkar,Christopher Zurenko,Emmanouil D. Karagiannis,Kevin T. Love,Delai Chen,Roberto Zoncu,Yosef Buganim,Avi Schroeder,Robert Langer,Daniel G. Anderson +13 more
TL;DR: In this paper, the authors examined cellular uptake of siRNA delivered in lipid nanoparticles (LNPs) using cellular trafficking probes in combination with automated high-throughput confocal microscopy as well as defined perturbations of cellular pathways paired with systems biology approaches to uncover protein-protein and protein-small molecule interactions.
Journal ArticleDOI
Progress Toward In Vivo Use of siRNAs-II
Garrett R. Rettig,Mark A. Behlke +1 more
TL;DR: Approval is placed on work with synthetic, small interfering RNAs (siRNAs) published since the first installment of this review which appeared in 2006, and on select publications that demonstrate interesting applications of these principles.
References
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Journal ArticleDOI
Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure
Philip L. Felgner,Thomas R. Gadek,Marilyn Holm,Richard Bolton Roman,Hardy W. Chan,Michael Wenz,Jeffrey P. Northrop,Gordon M. Ringold,Mark Danielsen +8 more
TL;DR: Depending upon the cell line, lipofection is from 5- to greater than 100-fold more effective than either the calcium phosphate or the DEAE-dextran transfection technique.
Journal ArticleDOI
Knocking down barriers: advances in siRNA delivery
TL;DR: An update on the progress of RNAi therapeutics is provided and novel synthetic materials for the encapsulation and intracellular delivery of nucleic acids are highlighted.
Journal ArticleDOI
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
Jürgen Soutschek,Akin Akinc,Birgit Bramlage,Klaus Charisse,Rainer Constien,Mary Donoghue,Sayda Elbashir,Anke Geick,Philipp Hadwiger,Jens Harborth,Matthias John,Venkitasamy Kesavan,Gary Lavine,Rajendra K. Pandey,Timothy Racie,Kallanthottathil G. Rajeev,Ingo Röhl,Ivanka Toudjarska,Gang Wang,Silvio Wuschko,David Bumcrot,Victor Koteliansky,Stefan Limmer,Muthiah Manoharan,Hans-Peter Vornlocher +24 more
TL;DR: In this article, chemically modified short interfering RNAs (siRNAs) were used to silence an endogenous gene encoding apolipoprotein B (apoB) after intravenous injection in mice.
Journal ArticleDOI
Rational design of cationic lipids for siRNA delivery
Sean C. Semple,Akin Akinc,Jianxin Chen,Ammen P. Sandhu,Barbara L. Mui,Connie K Cho,Dinah W.Y. Sah,Derrick Stebbing,Erin J Crosley,Ed Yaworski,Ismail M. Hafez,J. Robert Dorkin,June Qin,Kieu Lam,Kallanthottathil G. Rajeev,Kim F. Wong,Lloyd Jeffs,Lubomir Nechev,Merete L. Eisenhardt,Muthusamy Jayaraman,Mikameh Kazem,Martin Maier,Masuna Srinivasulu,Michael J Weinstein,Qingmin Chen,Rene Alvarez,Scott A Barros,Soma De,Sandra K. Klimuk,Todd Borland,Verbena Kosovrasti,William Cantley,Ying K. Tam,Muthiah Manoharan,Marco A. Ciufolini,Mark A Tracy,Antonin de Fougerolles,Ian MacLachlan,Pieter R. Cullis,Thomas D. Madden,Michael J. Hope +40 more
TL;DR: The best-performing lipid recovered after screening (DLin-KC2-DMA) was formulated and characterized in SNALP and demonstrated to have in vivo activity at siRNA doses as low as 0.01 mg/kg in rodents and 0.1 mg/ kg in nonhuman primates, a substantial improvement over previous reports of in vivo endogenous hepatic gene silencing.
Journal ArticleDOI
RNAi-mediated gene silencing in non-human primates
Tracy Zimmermann,Amy C.H. Lee,Akin Akinc,Birgit Bramlage,David Bumcrot,Matthew N. Fedoruk,Jens Harborth,James Heyes,Lloyd Jeffs,Matthias John,Adam Judge,Kieu Lam,Kevin McClintock,Lubomir Nechev,Lorne R. Palmer,Timothy Racie,Ingo Röhl,Stephan Seiffert,Sumi Shanmugam,Vandana Sood,Jürgen Soutschek,Ivanka Toudjarska,Amanda J. Wheat,Ed Yaworski,William Zedalis,Victor Koteliansky,Muthiah Manoharan,Hans-Peter Vornlocher,Ian MacLachlan +28 more
TL;DR: It is shown that siRNAs, when delivered systemically in a liposomal formulation, can silence the disease target apolipoprotein B in non-human primates, supporting RNAi therapeutics as a potential new class of drugs.
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