Hepatocyte-targeted RNAi Therapeutics for the Treatment of Chronic Hepatitis B Virus Infection
Christine I. Wooddell,David B. Rozema,Markus Hossbach,Matthias John,Holly Hamilton,Qili Chu,Julia Hegge,Jason Klein,Darren H. Wakefield,Claudia E. Oropeza,Jochen Deckert,Ingo Roehl,Kerstin Jahn-Hofmann,Philipp Hadwiger,Hans-Peter Vornlocher,Alan McLachlan,David L. Lewis +16 more
TLDR
It is shown that a single coinjection of NAG-MLP with potent chol-siRNAs targeting conserved HBV sequences resulted in multilog repression of viral RNA, proteins, and viral DNA with long duration of effect.About:
This article is published in Molecular Therapy.The article was published on 2013-05-01 and is currently open access. It has received 284 citations till now. The article focuses on the topics: RNAi Therapeutics & Viral load.read more
Citations
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Journal ArticleDOI
Non-viral vectors for gene-based therapy
Hao Yin,Rosemary Lynn Kanasty,Ahmed A. Eltoukhy,Arturo J. Vegas,J. Robert Dorkin,Daniel G. Anderson +5 more
TL;DR: The biological barriers to gene delivery in vivo are introduced and recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems are discussed, some of which are currently undergoing testing in clinical trials.
Journal ArticleDOI
Asian-Pacific clinical practice guidelines on the management of hepatitis B: a 2015 update.
Shiv Kumar Sarin,Manoj Kumar,George K. K. Lau,Zaigham Abbas,Henry Lik-Yuen Chan,Chien-Jen Chen,Ding-Shinn Chen,Huey-Ling Chen,Pei-Jer Chen,Rong-Nan Chien,Abdul Kadir Dokmeci,Edward Gane,J L Hou,Wasim Jafri,Jidong Jia,J. H. Kim,Ching-Lung Lai,Hon Cheung Lee,Seng Gee Lim,Chun-Jen Liu,Stephen Locarnini,M. Al Mahtab,Rosmawati Mohamed,Masao Omata,Jun Yong Park,Teerha Piratvisuth,Barjesh Chander Sharma,Jose D. Sollano,Fu-Sheng Wang,Lai Wei,Man-Fung Yuen,Shusen Zheng,Jia-Horng Kao +32 more
TL;DR: The final clinical practice guidelines and recommendations for the optimal management of chronic HBV infection are presented here, along with the relevant background information.
Journal ArticleDOI
Delivery materials for siRNA therapeutics
TL;DR: An introduction to the biological challenges that siRNA delivery materials aim to overcome is provided, as well as a discussion of the way that the most effective and clinically advanced classes of si RNA delivery systems are designed to surmount these challenges.
Journal ArticleDOI
The current state and future directions of RNAi-based therapeutics.
TL;DR: This Review discusses key advances in the design and development of RNAi drugs leading up to this landmark achievement, the state of the current clinical pipeline and prospects for future advances, including novel RNAi pathway agents utilizing mechanisms beyond post-translational RNAi silencing.
Journal ArticleDOI
Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
Jayaprakash K. Nair,Jennifer L. S. Willoughby,Amy Chan,Klaus Charisse,Md. Rowshon Alam,Qianfan Wang,Menno Hoekstra,Pachamuthu Kandasamy,Alexander V Kel'in,Stuart Milstein,Nate Taneja,Jonathan O'Shea,Sarfraz Shaikh,Ligang Zhang,Ronald J. van der Sluis,Michael E. Jung,Akin Akinc,Renta Hutabarat,Satya Kuchimanchi,Kevin Fitzgerald,Tracy Zimmermann,Theo J.C. Van Berkel,Martin Maier,Kallanthottathil G. Rajeev,Muthiah Manoharan +24 more
TL;DR: The optimally chemically modified siRNA-GalNAc conjugates are hepatotropic and long-acting and have the potential to treat a wide range of diseases involving liver-expressed genes.
References
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Book
Molecular Cloning: A Laboratory Manual
TL;DR: Molecular Cloning has served as the foundation of technical expertise in labs worldwide for 30 years as mentioned in this paper and has been so popular, or so influential, that no other manual has been more widely used and influential.
Journal ArticleDOI
Analysis of relative gene expression data using real-time quantitative pcr and the 2(-delta delta c(t)) method
TL;DR: The 2-Delta Delta C(T) method as mentioned in this paper was proposed to analyze the relative changes in gene expression from real-time quantitative PCR experiments, and it has been shown to be useful in the analysis of realtime, quantitative PCR data.
Journal ArticleDOI
Single-step method of RNA isolation by acid guanidinium thiocyanate-phenol-chloroform extraction
TL;DR: A new method of total RNA isolation by a single extraction with an acid guanidinium thiocyanate-phenol-chloroform mixture is described, providing a pure preparation of undegraded RNA in high yield and can be completed within 4 h.
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
Targeted disruption of the alpha isoform of the peroxisome proliferator-activated receptor gene in mice results in abolishment of the pleiotropic effects of peroxisome proliferators.
Susanna S.T. Lee,Thierry Pineau,John Drago,Eric J. Lee,Jennie W. Owens,Deanna L. Kroetz,Pedro M. Fernández-Salguero,Heiner Westphal,Frank J. Gonzalez +8 more
TL;DR: It is demonstrated that mPPAR alpha is the major isoform required for mediating the pleiotropic response resulting from the actions of peroxisome proliferators.
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