Target selectivity in mRNA silencing

Gene Therapy
N Aronin

Abstract

Despite the excitement and promise of RNA interference in treating neurodegenerative disease, disease gene mRNA might resist mRNA silencing. Conventional siRNA design does not uniformly distinguish a mutant from a wild-type allele. CAG expansions in trinucleotide repeat diseases are unselective targets for small siRNAs. This review will consider recent discoveries in mechanisms of RNA interference and siRNA modifications that improve siRNA selectivity, delivery and performance.

References

Jan 5, 2000·Genes & Development·T TuschlP A Sharp
Sep 17, 2002·Cell·Javier MartinezThomas Tuschl
Feb 13, 2003·Nature Medicine·Erwei SongJudy Lieberman
May 20, 2003·Nature Biotechnology·Aimee L JacksonPeter S Linsley
Jun 5, 2003·Proceedings of the National Academy of Sciences of the United States of America·Victor M MillerHenry L Paulson
Aug 19, 2003·RNA·Ya-Lin Chiu, Tariq M Rana
Aug 28, 2003·Nature Cell Biology·Carol A SledzBryan R G Williams
Oct 22, 2003·Cell·Dianne S SchwarzPhillip D Zamore
Oct 22, 2003·Cell·Anastasia KhvorovaSumedha D Jayasena
Feb 6, 2004·Nature Biotechnology·Angela ReynoldsAnastasia Khvorova
Feb 11, 2004·Proceedings of the National Academy of Sciences of the United States of America·Peter C ScacheriFrancis S Collins
Mar 12, 2004·Genes & Development·John G Doench, Phillip A Sharp
Mar 18, 2004·Nucleic Acids Research·Gabriele DornJonathan Hall
Jun 1, 2004·Nature Structural & Molecular Biology·Benjamin Haley, Phillip D Zamore
Jul 21, 2004·Molecular Cell·Gunter MeisterThomas Tuschl
Jul 31, 2004·Science·Ji-Joon SongLeemor Joshua-Tor
Jul 31, 2004·Science·Jidong LiuGregory J Hannon
Nov 13, 2004·The Journal of Neuroscience : the Official Journal of the Society for Neuroscience·Thomas J DavidsonCarol M Troy
Nov 13, 2004·Nature·Jürgen SoutschekHans-Peter Vornlocher
Nov 20, 2004·Science·Yukihide TomariPhillip D Zamore
Nov 24, 2004·Nature Biotechnology·Jeremy D HeidelMark E Davis
Dec 1, 2004·Proceedings of the National Academy of Sciences of the United States of America·Deepak R ThakkerJohn F Cryan
Dec 28, 2004·Nature Medicine·Vira BitkoSailen Barik
Jan 18, 2005·Nucleic Acids Research·Joacim ElménClaes Wahlestedt
Mar 3, 2005·Genes & Development·Yukihide Tomari, Phillip D Zamore

❮ Previous
Next ❯

Citations

Jul 20, 2007·Oligonucleotides·Ute KruegerJuliane Konrad
Oct 30, 2010·Oligonucleotides·Natalya S Petrova KruglovaElena L Chernolovskaya
Jun 16, 2011·Human Molecular Genetics·Daniel W ChungRussell L Margolis
Nov 22, 2011·International Journal of Pharmaceutics·Jian LiuLei Yu
Mar 18, 2009·Experimental Neurology·Maria Stella LombardiWilliam F Kaemmerer
Feb 24, 2007·Pharmacology & Therapeutics·Pedro Gonzalez-Alegre
Aug 29, 2014·Movement Disorders : Official Journal of the Movement Disorder Society·Neil Aronin, Marian DiFiglia
Dec 1, 2006·Journal of Clinical Neurology·Ichiro Kanazawa
Jul 29, 2010·Physiological Reviews·Chiara ZuccatoElena Cattaneo
Apr 8, 2017·Scientific Reports·A M OlivaresN B Haider
Mar 21, 2007·The Journal of Immunology : Official Journal of the American Association of Immunologists·Rana M Al-Sadi, Thomas Y Ma
May 21, 2019·Frontiers in Pharmacology·Ivan V ChernikovElena L Chernolovskaya

❮ Previous
Next ❯

Related Concepts

Related Feeds

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.