Get Advanced Delivery and Therapeutic Applications of RNAi PDF

By Kun Cheng, Ram I. Mahato

ISBN-10: 1118610741

ISBN-13: 9781118610749

ISBN-10: 1119976863

ISBN-13: 9781119976868

RNA interference (RNAi) is a procedure in dwelling cells wherein small double stranded RNA interferes with the expression of particular genes with complementary nucleotide series. Like many nucleic acid-based remedies, RNAi has nice capability in treating numerous life-threatening illnesses. even if, the negative balance and mobile uptake of RNAi molecules stay substantial obstacles to their effective supply that's paramount to a winning treatment. This e-book offers a complete advent to RNAi supply, then is going directly to speak about varied supply innovations, and concludes with present functions in numerous illnesses. It covers:

  • Mechanisms, boundaries, and research of RNAi Delivery
  • Nonclinical defense checks and scientific Pharmacokinetics: A Regulatory Perspective
  • Bioconjugation of siRNA for web site Specifi c Delivery
  • Nanoscale supply platforms for RNAi
  • Environmentally-Responsive supply platforms for RNAi
  • Light-sensitive RNAi
  • Viral-Mediated supply of shRNA and miRNA
  • RNAi functions in melanoma remedy, Liver illnesses, Hepatitis B, and Ocular Disease
  • miRNA as healing brokers and targets

Advanced supply and healing functions of RNAi combines the basic points of constructing RNAi therapeutics from bench to medical institution, and is precious for researchers engaged on RNAi, drug discovery and supply, biomedical engineering, biomaterials, molecular biology, and biotechnology.

Chapter 1 Mechanisms and limitations to RNAi supply (pages 3–17): Jiehua Zhou and John J. Rossi
Chapter 2 research of siRNA supply utilizing quite a few Methodologies (pages 19–44): Yi Pei
Chapter three demanding situations and possibilities in Bringing RNAi applied sciences from Bench to mattress (pages 45–62): Sandesh Subramanya and Lance Ford
Chapter four Nonclinical protection checks and medical Pharmacokinetics for Oligonucleotide Therapeutics: A Regulatory standpoint (pages 63–81): Shwu?Luan Lee, Paul Brown, Jian Wang and Robert T. Dorsam
Chapter five position of Promoters and Micro RNA spine for effective Gene Silencing (pages 83–108): Feng Li and Ram I. Mahato
Chapter 6 Bioconjugation of siRNA for Site?specific supply (pages 111–136): Bin Qin, Wei Jin and Kun Cheng
Chapter 7 Multifunctional RNAi supply structures (pages 137–161): China Malakondaiah Kummitha, Anthony S. Malamas and Zheng?Rong Lu
Chapter eight Dendrimers in RNAi supply (pages 163–185): Jose Luis Jimenez Fuentes, Paula Ortega, Sara Ferrando?Martinez, Rafael Gomez, Manuel Leal, Javier de los angeles Mata and M?Angeles Munoz? Fernandez
Chapter nine improvement of Pharmaceutically tailored Mesoporous Silica Nanoparticles for siRNA supply (pages 187–205): Wilson X. Mai, Tian Xia and Huan Meng
Chapter 10 Environmentally?Responsive Nanogels for siRNA supply (pages 207–224): Atsushi Tamura and Yukio Nagasaki
Chapter eleven Viral?Mediated supply of shRNA and miRNA (pages 225–254): Fredric P. Manfredsson
Chapter 12 The keep an eye on of RNA Interference with mild (pages 255–268): Simon H. Friedman
Chapter thirteen RNAi in melanoma remedy (pages 271–307): Cristian Rodriguez?Aguayo, Arturo Chavez?Reyes, Gabriel Lopez?Berestein and Anil ok. Sood
Chapter 18 Adenovirus?mediated shRNA supply to melanoma (pages 309–326): Chae?Ok Yun
Chapter 15 RNAi in Liver illnesses (pages 327–366): Jiang Li, Jianqin Lu, Yifei Zhang, Mohammed Ghazwani, Peng Zhang, Xiang Gao and tune Li
Chapter sixteen ways to providing RNAi Therapeutics that concentrate on Hepatitis B Virus (pages 367–389): Carol Crowther, Mohube Betty Mowa, Abdullah Ely and Patrick Arbuthnot
Chapter 17 RNAi in respiration ailments (pages 391–416): Ciara Kelly, Awadh B. Yadav, Paul J. McKiernan, Catherine M. Greene and Sally?Ann Cryan
Chapter 18 RNAi in Ocular ailments (pages 417–437): Andrey Turchinovich, Georg Zoidl and Rolf Dermietzel
Chapter 19 micro RNAs as healing brokers and pursuits (pages 439–482): D.S. Karolina and okay. Jeyaseelan
Chapter 20 supply of Micro RNA Sponges for Interrogation of Micro RNA functionality In Vitro and In Vivo (pages 483–504): Jiakai Lin and Shu Wang

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Extra resources for Advanced Delivery and Therapeutic Applications of RNAi

Sample text

91. Zhang, P. et al. (2009) Using an RNA aptamer probe for flow cytometry detection of CD30-expressing lymphoma cells. Laboratory Investigation, 89, 1423–1432. 92. Guo, S. et al. (2005) Specific delivery of therapeutic RNAs to cancer cells via the dimerization mechanism of phi29 motor pRNA. Human Gene Therapy, 16, 1097–1109. 93. Song, E. et al. (2003) RNA interference targeting Fas protects mice from fulminant hepatitis. Nature Medicine, 9, 347–351. 94. E. (2009) The first targeted delivery of siRNA in humans via a selfassembling, cyclodextrin polymer-based nanoparticle: from concept to clinic.

Journal of Molecular Graphics and Modelling, 25, 784–793. 19. S. et al. (2006) Comprehensive thermodynamic analysis of 30 doublenucleotide overhangs neighboring Watson-Crick terminal base pairs. Nucleic Acids Research, 34, 3338–3344. 20. S. et al. (2005) Stability of 30 double nucleotide overhangs that model the 30 ends of siRNA. RNA, 11, 512–516. 21. Zhang, H. et al. (2002) Human Dicer preferentially cleaves dsRNAs at their termini without a requirement for ATP. The EMBO Journal, 21, 5875–5885.

It has been demonstrated that hypermodification or improper positioning of the modification can compromise RNAi activity and even trigger cellular toxicity [46]. As an example, a siRNA with a boranophosphonate modification at the central position of the antisense strand resulted in improved stability but reduced the silencing potency [47]. 5 Cellular Uptake and Intracellular Release of siRNA Although it was recently reported that a minor pathway mediated by fusion between siRNA lipoplexes and the plasma membrane is responsible for functional siRNA delivery, 8 Advanced Delivery and Therapeutic Applications of RNAi the major pathway of internalizing a nanocarrier-mediated siRNA delivery system involves two subtypes of endocytosis [12,48,49]: phagocytosis and macropinocytosis.

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Advanced Delivery and Therapeutic Applications of RNAi by Kun Cheng, Ram I. Mahato

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