|
PMID |
Title of the article |
Author/s |
Journal Reference |
Year |
|
12242156 |
Stable
suppression of tumorigenicity by virus-mediated RNA interference. |
Brummelkamp TR, Bernards
R, Agami R |
Cancer
Cell. 2002 Sep;2(3):243-7 |
2002 |
|
11809726 |
Rescue of polyglutamine-mediated
cytotoxicity by double-stranded RNA-mediated RNA interference. |
Caplen NJ, Taylor JP, Statham VS, Tanaka F,
Fire A, Morgan RA. |
Hum Mol Genet. 2002 Jan 15;11(2):175-84. |
2002 |
|
11751575 |
HAT activity is
essential for CBP-1-dependent transcription and differentiation in Caenorhabditis elegans. |
Victor M, Bei Y, Gay F, Calvo D, Mello C,
Shi Y. |
EMBO
Rep. 2002 Jan;3(1):50-5. Epub
2001 Dec 19. |
2002 |
|
12403821 |
Synthetic small inhibiting RNAs:
Efficient tools to inactivate oncogenic mutations and restore p53 pathways. |
Martinez LA, Naguibneva
I, Lehrmann H, Vervisch
A, Tchenio T, Lozano G, Harel-Bellan A |
Proc Natl Acad Sci U S A. 2002 Nov
12;99(23):14849-54. Epub
2002 Oct 28 |
2002 |
|
26700961 |
Allele-specific
silencing of mutant p53 attenuates dominant- negative and gain-of-function
activities. |
Martinez
LA, Naguibneva I, Lehrmann
H, Vervisch A, Tchenio
T, Lozano G, Harel-Bellan A |
Proc Natl Acad Sci U S A. 2002 Nov
12;99(23):14849-54. Epub
2002 Oct 28 |
2002 |
|
12782788 |
Allele-specific silencing of dominant
disease genes. |
Miller VM, Xia H, Marrs
GL, Gouvion CM, Lee G, Davidson BL, Paulson HL. |
Proc Natl Acad Sci U S A. 2003 Jun 10;100(12):7195-200. Epub 2003 Jun
2. |
2003 |
|
12783425 |
Toward therapy
for DYT1 dystonia: allele-specific silencing of mutant TorsinA. |
Gonzalez-Alegre P, Miller VM, Davidson BL, Paulson HL |
Ann
Neurol. 2003 Jun;53(6):781-7 |
2003 |
|
12928480 |
Allele-specific silencing of a
pathogenic mutant acetylcholine receptor subunit by RNA interference. |
Abdelgany A, Wood M, Beeson D. |
Hum Mol Genet. 2003 Oct 15;12(20):2637-44.
Epub 2003 Aug 19. |
2003 |
|
12930974 |
An enhanced U6
promoter for synthesis of short hairpin RNA. |
Xia XG, Zhou H,
Ding H, Affar el B, Shi Y, Xu
Z |
Nucleic
Acids Res. 2003 Sep 1;31(17):e100 |
2003 |
|
12934714 |
Selective silencing by RNAi of a
dominant allele that causes amyotrophic lateral sclerosis. |
Ding H, Schwarz DS, Keene A, Affar el B, Fenton L, Xia X, Shi Y, Zamore
PD, Xu Z. |
Aging Cell.
2003 Aug;2(4):209-17. |
2003 |
|
12527766 |
Tolerance for
mutations and chemical modifications in a siRNA. |
Amarzguioui M, Holen T, Babaie E, Prydz H. |
Nucleic
Acids Res. 2003 Jan 15;31(2):589-95. |
2003 |
|
14754988 |
Targeting Alzheimer's disease genes
with RNA interference: an efficient strategy for silencing mutant alleles. |
Miller VM, Gouvion CM, Davidson BL,
Paulson HL. |
Nucleic Acids
Res. 2004 Jan 30;32(2):661-8. Print 2004. |
2004 |
|
15236410 |
Sequence-dependent
and independent inhibition specific for mutant ataxin-3 by small interfering RNA |
Li Y, Yokota T,
Matsumura R, Taira K, Mizusawa
H |
Ann
Neurol. 2004 Jul;56(1):124-9 |
2004 |
|
14981234 |
RNA interference-mediated silencing of
mutant superoxide dismutase rescues cyclosporin A-induced death in cultured neuroblastoma cells. |
Maxwell MM, Pasinelli
P, Kazantsev AG, Brown RH Jr |
Proc Natl Acad Sci U S A. 2004 Mar 2;101(9):3178-83. Epub 2004 Feb 23 |
2004 |
|
15241481 |
RNAi of COL1A1 in mesenchymal
progenitor cells. |
Millington-Ward
S, McMahon HP, Allen D, Tuohy G, Kiang AS, Palfi A, Kenna PF, Humphries P,
Farrar GJ. |
Eur J Hum Genet. 2004 Oct;12(10):864-6 |
2004 |
|
14741366 |
Enhancement of RNAi activity by
improved siRNA duplexes. |
Hohjoh H. |
FEBS Lett. 2004 Jan 16;557(1-3):193-8. |
2004 |
|
16280588 |
Silencing
primary dystonia: lentiviral-mediated RNA
interference therapy for DYT1 dystonia. |
Gonzalez-Alegre P, Bode N, Davidson BL, Paulson HL |
J
Neurosci. 2005 Nov 9;25(45):10502-9 |
2005 |
|
16019264 |
Intrastriatal rAAV-mediated
delivery of anti-huntingtin shRNAs induces partial
reversal of disease progression in R6/1 Huntington's disease transgenic mice. |
Rodriguez-Lebron
E, Denovan-Wright EM, Nash K, Lewin
AS, Mandel RJ |
Mol Ther. 2005
Oct;12(4):618-33 |
2005 |
|
16225852 |
Selective
silencing of a mutant transthyretin
allele by small interfering RNAs. |
Kurosawa T,
Igarashi S, Nishizawa M, Onodera O |
Biochem Biophys Res Commun. 2005 Nov 25;337(3):1012-8.
Epub 2005 Oct 3 |
2005 |
|
16113241 |
Tolerated wobble mutations in siRNAs
decrease specificity, but can enhance activity in vivo. |
Holen T, Moe SE, Sżrbż
JG, Meza TJ, Ottersen OP, Klungland
A. |
Nucleic Acids
Res. 2005 Aug 19;33(15):4704-10. Print 2005. |
2005 |
|
US2005/0176045A1 |
SNP
discriminatory siRNA |
Yuriy Fedorov,
Angela Reynolds, Anastasia Khvorova |
US20050176045
A1 |
2005 |
|
US20080176812A1 |
Allele-specific silencing of disease
genes. |
Davidson Beverly L, Harper Scott,
Gonzalez-Alegre Pedro,Miller Victor, Paulson Henry |
US2008/0176812
A1 |
2005 |
|
15781493 |
A systematic
analysis of the silencing effects of an active siRNA at all single-nucleotide
mismatched target sites. |
Du Q, Thonberg H, Wang J, Wahlestedt
C, Liang Z. |
Nucleic
Acids Res. 2005 Mar 21;33(5):1671-7. Print 2005.
Erratum in: Nucleic Acids Res. 2005;33(11):3698. |
2005 |
|
19771217 |
Assessment of allele-specific gene
silencing by RNA interference with mutant and wild-type
reporter alleles. |
Ohnishi Y, Tokunaga K, Kaneko K, Hohjoh
H. |
J RNAi Gene
Silencing. 2006 Feb 28;2(1):154-60. |
2006 |
|
16857362 |
Allele-specific
RNAi selectively silences mutant SOD1
and achieves significant therapeutic benefit in vivo. |
Xia X, Zhou H,
Huang Y, Xu Z |
Neurobiol Dis. 2006 Sep;23(3):578-86.
Epub 2006 Jul 20 |
2006 |
|
16455076 |
Silencing of human alpha-synuclein in vitro and in rat brain using lentiviral-mediated RNAi. |
Sapru MK, Yates JW, Hogan S, Jiang L, Halter
J, Bohn MC. |
Exp Neurol. 2006 Apr;198(2):382-90.
Epub 2006 Feb 7. |
2006 |
|
16426772 |
Allele-specific
silencing of Alzheimer's disease genes: the amyloid precursor protein genes
with Swedish or London mutations. |
Feng X, Zhao P, He Y, Zuo
Z. |
Gene.
2006 Apr 12;371(1):68-74. Epub
2006 Jan 19. |
2006 |
|
16965178 |
Designing siRNA that distinguish
between genes that differ by a single nucleotide. |
Schwarz DS, Ding H, Kennington
L, Moore JT, Schelter J, Burchard
J, Linsley PS, Aronin N, Xu Z, Zamore PD. |
PLoS Genet. 2006 Sep 8;2(9):e140.
Epub 2006 Jul 24. |
2006 |
|
16520945 |
Class-, gene-,
and group-specific HLA silencing by lentiviral
shRNA delivery. |
Figueiredo C, Seltsam
A, Blasczyk R. |
J
Mol Med (Berl). 2006 May;84(5):425-37. Epub 2006 Mar 7. |
2006 |
|
16585504 |
Determinants of specific RNA
interference-mediated silencing of human beta-globin alleles differing by a
single nucleotide polymorphism. |
Dykxhoorn DM, Schlehuber
LD, London IM, Lieberman J. |
Proc Natl Acad Sci U S A. 2006 Apr 11;103(15):5953-8. Epub 2006 Apr
3. |
2006 |
|
17145926 |
SiRNA-mediated
selective inhibition of mutant keratin mRNAs responsible for the skin
disorder pachyonychia congenita. |
Hickerson RP, Smith FJ, McLean WH, Landthaler M, Leube RE, Kaspar RL. |
Ann
N Y Acad Sci. 2006 Oct;1082:56-61. |
2006 |
|
16489337 |
3' UTR seed matches, but not overall
identity, are associated with RNAi off-targets. |
Birmingham A, Anderson EM, Reynolds A, Ilsley-Tyree D, Leake D, Fedorov Y, Baskerville S, Maksimova
E, Robinson K, Karpilow J, Marshall WS, Khvorova A. |
Nat Methods.
2006 Mar;3(3):199-204 |
2006 |
|
US2007/0259827A1 |
Compositions
And Methods For Enhancing Discriminatory Rna Interference |
Neil Aronin, Dianne Schwarz, Phillip D. Zamore |
US2007/0259827A1 |
2007 |
|
18493311 |
Enhancement of allele discrimination by
introduction of nucleotide mismatches into siRNA in allele-specific gene
silencing by RNAi. |
Ohnishi Y, Tamura Y, Yoshida M,
Tokunaga K, Hohjoh H. PLoS One. |
2008 May 21;3(5):e2248. doi:
10.1371/journal.pone.0002248. |
2008 |
|
18653764 |
Allele-specific
silencing of the dominant disease allele in sialuria by RNA interference |
Klootwijk RD, Savelkoul
PJ, Ciccone C, Manoli I, Caplen NJ, Krasnewich DM, Gahl WA, Huizing M |
FASEB
J. 2008 Nov;22(11):3846-52. doi: 10.1096/fj.08-110890. Epub
2008 Jul 24 |
2008 |
|
18841197 |
Allele-Specific RNA Silencing of Mutant
Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease. |
Alves S, Nascimento-Ferreira
I, Auregan G, Hassig R, Dufour N, Brouillet E, Pedroso de Lima MC, Hantraye P,
Pereira de Almeida L, DŽglon N |
PLoS One. 2008 Oct 8;3(10):e3341.
doi:
10.1371/journal.pone.0003341 |
2008 |
|
19015742 |
Allele
dependent silencing of COL1A2 using
small interfering RNAs. |
Lindahl K, Rubin CJ, Kindmark
A, Ljunggren O. |
Int J Med Sci. 2008;5(6):361-5.
Epub 2008 Nov 12. |
2008 |
|
18931668 |
Linking SNPs to CAG repeat length in
Huntington's disease patients. |
Liu W, Kennington
LA, Rosas HD, Hersch S, Cha JH, Zamore
PD, Aronin N |
Nat Methods.
2008 Nov;5(11):951-3. doi: 10.1038/nmeth.1261. Epub
2008 Oct 19 |
2008 |
|
18776243 |
RNA
interference in silencing of genes of Alzheimer's disease in cellular and rat
brain models. |
Sierant M, Kubiak K,
Kazmierczak-Baranska J, Paduszynska
A, Kuwabara T, Warashina
M, Nacmias B, Sorbi S, Nawrot B |
Nucleic
Acids Symp Ser (Oxf). 2008;(52):41-2. doi: 10.1093/nass/nrn021 |
2008 |
|
17914454 |
Single-nucleotide-specific siRNA
targeting in a dominant-negative skin model. |
Hickerson RP, Smith FJ, Reeves RE, Contag CH, Leake D, Leachman SA, Milstone LM,
McLean WH, Kaspar RL. |
J Invest Dermatol. 2008 Mar;128(3):594-605.
Epub 2007 Oct 11. |
2008 |
|
18258738 |
siRNA knock-down of mutant torsinA
restores processing through secretory pathway in DYT1 dystonia cells. |
Hewett JW, Nery FC, Niland B, Ge P, Tan P, Hadwiger P, Tannous BA, Sah DW, Breakefield XO |
Hum
Mol Genet. 2008 May 15;17(10):1436-45.
doi: 10.1093/hmg/ddn032. Epub 2008 Feb 7 |
2008 |
|
18215350 |
Double-mismatched siRNAs enhance
selective gene silencing of a mutant ALS-causing allele. |
Geng CM, Ding HL. |
Acta Pharmacol
Sin. 2008 Feb;29(2):211-6. doi: 10.1111/j.1745-7254.2008.00740.x. |
2008 |
|
18420656 |
Analysis of
siRNA specificity on targets with double-nucleotide mismatches. |
Dahlgren C,
Zhang HY, Du Q, Grahn M, Norstedt
G, Wahlestedt C, Liang Z. |
Nucleic
Acids Res. 2008 May;36(9):e53. doi: 10.1093/nar/gkn190. Epub 2008 Apr 17. |
2008 |
|
17762855 |
Development of Therapeutic siRNAs for
Pachyonychia Congenita |
Smith FJ, Hickerson
RP, Sayers JM, Reeves RE, Contag CH, Leake D, Kaspar RL, McLean WH |
J Invest Dermatol. 2008 Jan;128(1):50-8. Epub 2007 Aug 30 |
2008 |
|
19289118 |
A majority of
Huntington's disease patients may be treatable by individualized
allele-specific RNA interference. |
Lombardi MS,
Jaspers L, Spronkmans C, Gellera
C, Taroni F, Di Maria E, Donato
SD, Kaemmerer WF. |
Exp Neurol. 2009 Jun;217(2):312-9.
doi: 10.1016/j.expneurol.2009.03.004.
Epub 2009 Mar 13. |
2009 |
|
19532137 |
Allele-specific RNAi mitigates
phenotypic progression in a transgenic model of Alzheimer's disease. |
Rodr’guez-Lebr—n
E, Gouvion CM, Moore SA, Davidson BL, Paulson HL. |
Mol Ther. 2009
Sep;17(9):1563-73. doi: 10.1038/mt.2009.123. Epub
2009 Jun 16. |
2009 |
|
19094060 |
Allele-specific
silencing of mutant Huntington's disease gene |
Zhang Y, Engelman J, Friedlander RM |
J
Neurochem. 2009 Jan;108(1):82-90.
doi: 10.1111/j.1471-4159.2008.05734.x |
2009 |
|
15768029 |
Five siRNAs targeting three SNPs may
provide therapy for three-quarters of Huntington's disease patients. |
Pfister EL, Kennington
L, Straubhaar J, Wagh S,
Liu W, DiFiglia M, Landwehrmeyer
B, Vonsattel JP, Zamore
PD, Aronin N. |
Curr Biol. 2009 May 12;19(9):774-8.
doi:
10.1016/j.cub.2009.03.030. Epub 2009 Apr 9. |
2009 |
|
19789634 |
Design of RNAi
hairpins for mutation-specific silencing of ataxin-7 and correction of a SCA7
phenotype. |
Scholefield J, Greenberg LJ, Weinberg MS,
Arbuthnot PB, Abdelgany A, Wood MJ. |
PLoS One. 2009 Sep 30;4(9):e7232.
doi:
10.1371/journal.pone.0007232. |
2009 |
|
19165236 |
Allele-specific
cancer cell killing in vitro and in vivo targeting a single-nucleotide
polymorphism in POLR2A. |
Mook OR, Baas F, de Wissel
MB, Fluiter K. |
Cancer Gene Ther. 2009 Jun;16(6):532-8. doi: 10.1038/cgt.2008.104. Epub 2009 Jan 23. |
2009 |
|
US007576197B2 |
SiRNA targeting KRAS |
Anastasia Khvorova, Angela Reynolds, Devin Leake, William Marshall, Stephen Scaringe,Steven Read |
US007576197B2 |
2009 |
|
19935778 |
First-in-human Mutation-targeted siRNA
Phase Ib Trial of an Inherited Skin Disorder |
Leachman SA, Hickerson
RP, Schwartz ME, Bullough EE, Hutcherson SL, Boucher KM, Hansen CD, Eliason MJ, Srivatsa GS,Kornbrust DJ, Smith
FJ, McLean WI, Milstone LM, Kaspar RL |
Mol Ther. 2010
Feb;18(2):442-6. doi: 10.1038/mt.2009.273. Epub
2009 Nov 24 |
2009 |
|
EP1567539B1 |
Allele-specific
Rna Interference |
Xu Zuoshang,Zamore Phillip D |
EP1567539B1 |
2009 |
|
19815667 |
Profiling of mismatch discrimination in
RNAi enabled rational design of allele-specific siRNAs. |
Huang H, Qiao
R, Zhao D, Zhang T, Li Y, Yi F, Lai F, Hong J, Ding X, Yang Z, Zhang L, Du Q,
Liang Z. |
Nucleic Acids
Res. 2009 Dec;37(22):7560-9. doi: 10.1093/nar/gkp835. Epub . |
2009 |
|
21095568 |
Allele-selective
inhibition of huntingtin expression by switching to
an miRNA-like RNAi mechanism. |
Hu J, Liu J,
Corey DR. |
Chem Biol. 2010 Nov 24;17(11):1183-8.
doi:
10.1016/j.chembiol.2010.10.013. |
2010 |
|
22174841 |
Development of allele-specific
therapeutic siRNA in Meesmann epithelial corneal dystrophy. |
Liao H, Irvine AD, Macewen CJ, Weed KH,
Porter L, Corden LD, Gibson AB, Moore JE, Smith FJ, McLean WH, Moore CB. |
PLoS One. 2011;6(12):e28582.
doi:
10.1371/journal.pone.0028582. Epub 2011 Dec 12. |
2011 |
|
22031823 |
Identification
of allele-specific RNAi effectors targeting genetic forms of Parkinson's
disease. |
Sibley CR, Wood
MJ. |
PLoS One. 2011;6(10):e26194.
doi:
10.1371/journal.pone.0026194. Epub 2011 Oct 21. |
2011 |
|
21559198 |
Specific Silencing of L392V PSEN1
Mutant Allele by RNA Interference. |
Sierant M, Paduszynska
A, Kazmierczak-Baranska J, Nacmias
B, Sorbi S, Bagnoli S, Sochacka E, Nawrot B. |
Int J Alzheimers
Dis. 2011 Apr 7;2011:809218. doi: 10.4061/2011/809218. |
2011 |
|
21716320 |
Development of
allele-specific therapeutic siRNA for keratin 5 mutations in epidermolysis bullosa simplex. |
Atkinson SD, McGilligan VE, Liao H, Szeverenyi
I, Smith FJ, Moore CB, McLean WH. |
J
Invest Dermatol. 2011 Oct;131(10):2079-86.
doi: 10.1038/jid.2011.169.
Epub 2011 Jun 30. |
2011 |
|
21712955 |
Efficient allele-specific targeting of
LRRK2 R1441 mutations mediated by RNAi. |
de Y–igo-Mojado
L, Mart’n-Ru’z I, Sutherland JD. |
PLoS One. 2011;6(6):e21352.
doi:
10.1371/journal.pone.0021352. Epub 2011 Jun 21. |
2011 |
|
21364589 |
Activated
KrasG¹²D is associated with invasion and metastasis of pancreatic
cancer cells through inhibition of E-cadherin |
Rachagani S, Senapati
S, Chakraborty S, Ponnusamy
MP, Kumar S, Smith LM, Jain M, Batra SK |
Br
J Cancer. 2011 Mar 15;104(6):1038-48. doi: 10.1038/bjc.2011.31. Epub 2011 Mar 1 |
2011 |
|
21427085 |
Inhibition of mutant huntingtin expression by RNA duplex targeting expanded
CAG repeats. |
Fiszer A, Mykowska
A, Krzyzosiak WJ. |
Nucleic Acids
Res. 2011 Jul;39(13):5578-85. doi: 10.1093/nar/gkr156. Epub 2011 Mar 22. |
2011 |
|
21294677 |
Allele-selective
inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex
RNAs |
Hu J, Gagnon
KT, Liu J, Watts JK, Syeda-Nawaz J, Bennett CF,
Swayze EE, Randolph J, Chattopadhyaya J, Corey DR |
Biol Chem. 2011 Apr;392(4):315-25.
doi: 10.1515/BC.2011.045. Epub 2011 Feb 7 |
2011 |
|
23152933 |
Allele-specific gene silencing in two
mouse models of autosomal dominant skeletal myopathy. |
Loy RE, Lueck
JD, Mostajo-Radji MA, Carrell
EM, Dirksen RT. |
PLoS One. 2012;7(11):e49757.
doi:
10.1371/journal.pone.0049757. Epub 2012 Nov 12. |
2012 |
|
22336946 |
siRNA-mediated allele-specific inhibition of
mutant type VII collagen in dominant dystrophic epidermolysis bullosa. |
Pendaries V, Gasc G, Titeux M, Tonasso L, Mej’a JE, Hovnanian A. |
J
Invest Dermatol. 2012 Jun;132(6):1741-3.
doi: 10.1038/jid.2012.11. Epub 2012 Feb 16. |
2012 |
|
22038052 |
Allele-specific siRNA knockdown as a
personalized treatment strategy for vascular Ehlers-Danlos
syndrome in human fibroblasts. |
Mźller GA, Hansen U, Xu Z, Griswold B, Talan MI,
McDonnell NB, Briest W. |
FASEB J. 2012
Feb;26(2):668-77. doi: 10.1096/fj.11-182162. Epub
2011 Oct 28. |
2012 |
|
22130450 |
Disease-causing
allele-specific silencing against the ALK2 mutants, R206H and G356D, in fibrodysplasia ossificans progressiva. |
Takahashi M, Katagiri T, Furuya H, Hohjoh H. |
Gene
Ther. 2012 Jul;19(7):781-5.
doi: 10.1038/gt.2011.193. Epub 2011 Dec 1. |
2012 |
|
22402445 |
Generic and personalized RNAi-based
therapeutics for a dominant-negative epidermal fragility disorder. |
Leslie Pedrioli
DM, Fu DJ, Gonzalez-Gonzalez E, Contag CH, Kaspar RL, Smith FJ, McLean WH. |
J Invest Dermatol. 2012 Jun;132(6):1627-35.
doi: 10.1038/jid.2012.28. Epub 2012 Mar 8. |
2012 |
|
23042244 |
Mechanism of
allele-selective inhibition of huntingtin
expression by duplex RNAs that target CAG repeats: function through the RNAi
pathway. |
Hu J, Liu J, Yu
D, Chu Y, Corey DR. |
Nucleic
Acids Res. 2012 Dec;40(22):11270-80. doi: 10.1093/nar/gks907.
Epub 2012 Oct 4. |
2012 |
|
22939619 |
Single-stranded RNAs use RNAi to
potently and allele-selectively inhibit mutant huntingtin
expression. |
Yu D, Pendergraff
H, Liu J, Kordasiewicz HB, Cleveland DW, Swayze EE,
Lima WF, Crooke ST, Prakash TP, Corey DR. |
Cell. 2012 Aug
31;150(5):895-908. doi: 10.1016/j.cell.2012.08.002. |
2012 |
|
22397573 |
An evaluation
of oligonucleotide-based therapeutic strategies for polyQ
diseases. |
Fiszer A, Olejniczak
M, Switonski PM, Wroblewska
JP, Wisniewska-Kruk J, Mykowska
A, Krzyzosiak WJ. |
BMC
Mol Biol. 2012 Mar 7;13:6.
doi:
10.1186/1471-2199-13-6. |
2012 |
|
23349684 |
Silencing mutant ataxin-3 rescues motor
deficits and neuropathology in Machado-Joseph disease transgenic mice |
N—brega C, Nascimento-Ferreira
I, Onofre I, Albuquerque D, Hirai H, DŽglon N, de Almeida LP |
PLoS One. 2013;8(1):e52396.
doi:
10.1371/journal.pone.0052396. Epub 2013 Jan 22 |
2013 |
|
23233254 |
Allele-specific
siRNA silencing for the common keratin 12 founder mutation in Meesmann
epithelial corneal dystrophy. |
Allen EH,
Atkinson SD, Liao H, Moore JE, Leslie Pedrioli DM,
Smith FJ, McLean WH, Moore CB. |
Invest
Ophthalmol Vis Sci. 2013 Jan 17;54(1):494-502.
doi:
10.1167/iovs.12-10528. |
2013 |
|
24092743 |
Allele-specific silencing of mutant
Myh6 transcripts in mice suppresses hypertrophic cardiomyopathy. |
Jiang J, Wakimoto
H, Seidman JG, Seidman
CE. |
Science. 2013
Oct 4;342(6154):111-4. doi: 10.1126/science.1236921. |
2013 |
|
23951344 |
Specific
inhibition of tumor cells by oncogenic EGFR specific silencing by RNA interference. |
Takahashi M, Chiyo T, Okada T, Hohjoh H. |
PLoS One. 2013 Aug 8;8(8):e73214.
doi:
10.1371/journal.pone.0073214. eCollection
2013. |
2013 |
|
23470493 |
Allele-specific RNA interference
rescues the long-QT syndrome phenotype in human-induced pluripotency
stem cell cardiomyocytes |
Matsa E, Dixon JE, Medway C, Georgiou O,
Patel MJ, Morgan K, Kemp PJ, Staniforth A, Mellor
I, Denning C |
Eur Heart J. 2014 Apr;35(16):1078-87.
doi: 10.1093/eurheartj/eht067. Epub 2013 Mar
6 |
2014 |
|
24022296 |
Allele-specific
Col1a1 silencing reduces mutant collagen in fibroblasts from Brtl mouse, a model for classical osteogenesis
imperfecta. |
Rousseau J, Gioia R, Layrolle P, Lieubeau B, Heymann D, Rossi A,
Marini JC, Trichet V, Forlino
A. |
Eur J Hum Genet. 2014 May;22(5):667-74. doi:
10.1038/ejhg.2013.198. Epub 2013 Sep 11. |
2014 |
|
24355919 |
Antisense oligonucleotide treatment
ameliorates alpha-1 antitrypsin-related liver disease in mice |
Guo S, Booten
SL, Aghajan M, Hung G, Zhao C, Blomenkamp
K, Gattis D, Watt A, Freier
SM, Teckman JH, McCaleb
ML, Monia BP |
J Clin Invest. 2014 Jan;124(1):251-61.
doi: 10.1172/JCI67968. Epub 2013 Dec 20 |
2014 |
|
24518369 |
siRNA-mediated Allele-specific Silencing of
a COL6A3 Mutation in a Cellular Model of Dominant Ullrich
Muscular Dystrophy. |
Bolduc V, Zou Y, Ko D, Bšnnemann CG. |
Mol Ther Nucleic
Acids. 2014 Feb 11;3:e147. doi: 10.1038/mtna.2013.74. |
2014 |
|
24694346 |
Exploring the effect of sequence length
and composition on allele-selective inhibition of human huntingtin
expression by single-stranded silencing RNAs. |
Hu J, Liu J, Yu D, Aiba
Y, Lee S, Pendergraff H, Boubaker
J, Artates JW, Lagier-Tourenne
C, Lima WF, Swayze EE, Prakash TP, Corey DR. |
Nucleic Acid Ther. 2014 Jun;24(3):199-209. doi: 10.1089/nat.2013.0476. Epub 2014 Apr 2 |
2014 |
|
24801514 |
siRNA silencing of the mutant keratin 12
allele in corneal limbal epithelial cells grown
from patients with Meesmann's epithelial corneal
dystrophy |
Courtney DG,
Atkinson SD, Allen EH, Moore JE, Walsh CP, Pedrioli
DM, MacEwen CJ, Pellegrini
G, Maurizi E, Serafini C,
Fantacci M, Liao H, Irvine AD, McLean WH, Moore CB. |
Invest
Ophthalmol Vis Sci. 2014 May 6;55(5):3352-60.
doi: 10.1167/iovs.13-12957 |
2014 |
|
24926995 |
Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells. |
Drouet V, Ruiz M, Zala
D, Feyeux M, Auregan G, Cambon K, Troquier L, Carpentier J, Aubert S, Merienne N, Bourgois-Rocha F, Hassig R, Rey M, Dufour N, Saudou F, Perrier AL, Hantraye
P, DŽglon N. |
PLoS One. 2014 Jun 13;9(6):e99341.
doi:
10.1371/journal.pone.0099341. eCollection
2014 |
2014 |
|
24959844 |
Allele-specific
Gene Silencing of Mutant mRNA Restores Cellular Function in Ullrich Congenital Muscular Dystrophy Fibroblasts. |
Noguchi S,
Ogawa M, Kawahara G, Malicdan MC, Nishino I. |
Mol Ther Nucleic
Acids. 2014 Jun 24;3:e171. doi: 10.1038/mtna.2014.22 |
2014 |
|
24981774 |
Allele-selective inhibition of mutant
atrophin-1 expression by duplex and single-stranded RNAs. |
Hu J, Liu J, Narayanannair
KJ, Lackey JG, Kuchimanchi S, Rajeev KG, Manoharan M, Swayze EE, Lima WF, Prakash
TP, Xiang Q, Martinez C, Corey DR |
Biochemistry.
2014 Jul 22;53(28):4510-8. doi: 10.1021/bi500610r. Epub
2014 Jul 11 |
2014 |
|
24425855 |
Development of
allele-specific gene-silencing siRNAs for TGFBI Arg124Cys in lattice corneal
dystrophy type I |
Courtney
DG, Atkinson SD, Moore JE, Maurizi
E, Serafini C, Pellegrini
G, Black GC, Manson FD, Yam GH, Macewen CJ, Allen
EH, McLean WH, Moore CB |
Invest
Ophthalmol Vis Sci. 2014 Feb 18;55(2):977-85. doi:
10.1167/iovs.13-13279 |
2014 |
|
US8859752B2 |
Sirna-based Therapy Of Fibrodyplasia Ossificans Progressiva (fop) |
Kaplan Frederick S, Shore Eileen M |
US8859752B2 |
2014 |
|
25965551 |
Normalization
of Overexpressed α-Synuclein Causing
ParkinsonŐs Disease By a Moderate Gene Silencing With RNA Interference |
Takahashi M,
Suzuki M, Fukuoka M, Fujikake N, Watanabe S, Murata
M, Wada K, Nagai Y, Hohjoh H |
Mol Ther Nucleic
Acids. 2015 May 12;4:e241. doi: 10.1038/mtna.2015.14 |
2015 |
|
25849618 |
Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In
Vivo |
Monteys AM,Wilson MJ,Boudreau RL,Spengler RM,Davidson BL |
Mol Ther Nucleic
Acids. 2015 Apr 7;4:e234. doi: 10.1038/mtna.2015.7 |
2015 |
|
25586224 |
Efficient
CRISPR-rAAV engineering of endogenous genes to
study protein function by allele-specific RNAi |
Kaulich M, Lee YJ, Lšnn
P, Springer AD, Meade BR, Dowdy SF |
Nucleic
Acids Res. 2015 Apr 20;43(7):e45. doi: 10.1093/nar/gku1403.
Epub 2015 Jan 13 |
2015 |
|
27195674 |
Allele-specific silencing of EEC p63
mutant R304W restores p63 transcriptional activity |
Novelli F, Lena AM, Panatta
E, Nasser W, Shalom-Feuerstein R, Candi E, Melino G. |
Cell Death
Dis. 2016 May 19;7:e2227. doi: 10.1038/cddis.2016.118 |
2016 |
|
26891374 |
Correction of
mutant p63 in EEC syndrome using siRNA mediated allele specific silencing
restores defective stem cell function |
Barbaro V, Nasti AA,
Del Vecchio C, Ferrari S, Migliorati
A, Raffa P, Lariccia V, Nespeca P, Biasolo M, Willoughby
CE4, Ponzin D, Palť G, Parolin C, Di Iorio E,. |
Stem
Cells. 2016 Feb 17. doi:
10.1002/stem.2343 |
2016 |
|
26763422 |
Correction of Hair Shaft Defects
through Allele-Specific Silencing of Mutant Krt75 |
Liu Y, Snedecor
ER, Zhang X, Xu Y, Huang L, Jones EC, Zhang L,
Clark RA, Roop DR, Qin C, Chen J |
J Invest Dermatol. 2016 Jan;136(1):45-51.
doi: 10.1038/JID.2015.375 |
2016 |
|
27003758 |
A Small Indel Mutant Mouse Model of Epidermolytic
Palmoplantar Keratoderma
and Its Application to Mutant-specific shRNA Therapy |
Ya-Su Lyu, Pei-liang Shi, Xiao-Ling Chen, Yue-Xiao
Tang, Yan-Fang Wang, Rong-Rong Liu, Xiao-Rui Luan, Yu Fang4, Ru-Huan
Mei4 Zhen-Fang Du, Hai-Ping Ke5, Erik Matro6,
Ling-En Li, Zhao-Yu Lin, Jing Zhao, Xiang Gao and
Xian-Ning Zhang |
Mol Ther Nucleic
Acids. 2016 Mar 22;5:e299. doi: 10.1038/mtna.2016.17 |
2016 |
|
27003755 |
Design, Characterization, and Lead
Selection of Therapeutic miRNAs Targeting Huntingtin
for Development of Gene Therapy for Huntington's Disease |
Miniarikova J,, Zanella I, Huseinovic A, van
der Zon T, Hanemaaijer E,
Martier R,, Koornneef A, Southwell AL, Hayden MR, van Deventer SJ, Petry H, Konstantinova P. |
Mol Ther Nucleic
Acids. 2016 Mar 22;5:e297. doi: 10.1038/mtna.2016.7. |
2016 |