Biomedical Engineering Reference
In-Depth Information
neurological symptoms by an intracellular
antibody implicates the cytoplasmic toxicity of
mutant huntingtin. J Cell Biol 181(5):
803-816
62. Qin Z, Wang Y, Sapp E et al (2004) Huntingtin
bodies sequester vesicle-associated proteins by a
polyproline-dependent interaction. J Neurosci
24(1):269-281
63. Southwell AL, Khoshnan A, Dunn D et al
(2008) Intrabodies binding the proline-rich
domains of mutant huntingtin increase its
turnover and reduce neurotoxicity. J Neurosci
28(36):9013-9020
64. Southwell AL, Ko J, Patterson PH (2009)
Intrabody gene therapy ameliorates motor,
cognitive and neuropathological symptoms in
multiple mouse models of Huntington's dis-
ease. J Neurosci 29(43):13589-13602
65. Almeida R, Allshire RC (2005) RNA silencing
and genome regulation. Trends Cell Biol
15(5):251-258
66. Bartel DP (2004) MicroRNAs: genomics, bio-
genesis, mechanism, and function. Cell
116(2):281-297
67. Yamamoto A, Lucas JJ, Hen R (2000) Reversal
of neuropathology and motor dysfunction in a
conditional model of Huntington's disease.
Cell 101(1):57-66
68. Paddison PJ, Caudy AA, Bernstein E et al
(2002) Short hairpin RNAs (shRNAs) induce
sequence-specific silencing in mammalian cells.
Genes Dev 16(8):948-958
69. Harper SQ, Staber PD, He X et al (2005) RNA
interference improves motor and neuropatho-
logical abnormalities in a Huntington's disease
mouse model. Proc Natl Acad Sci U S A
102(16):5820-5825
70. Rodriguez-Lebron E, Denovan-Wright EM,
Nash K et al (2005) Intrastriatal rAAV-
mediated delivery of anti-huntingtin shRNAs
induces partial reversal of disease progression
in R6/1 Huntington's disease transgenic mice.
Mol Ther 12(4):618-633
71. Luthi-Carter R, Hanson SA, Strand AD et al
(2002) Dysregulation of gene expression in the
R6/2 model of polyglutamine disease: parallel
changes in muscle and brain. Hum Mol Genet
11(17):1911-1926
72. Machida Y, Okada T, Kurosawa M et al (2006)
rAAV-mediated shRNA ameliorated neuropa-
thology in Huntington disease model mouse.
Biochem Biophys Res Commun 343(1):
190-197
73. Franich NR, Fitzsimons HL, Fong DM et al
(2008) AAV vector-mediated RNAi of mutant
huntingtin expression is neuroprotective in a
novel genetic rat model of Huntington's dis-
ease. Mol Ther 16(5):947-956
74. Huang B, Schiefer J, Sass C et al (2007) High-
capacity adenoviral vector-mediated reduction
of huntingtin aggregate load in vitro and
in vivo. Hum Gene Ther 18(4):303-311
75. Drouet V, Perrin V, Hassig R et al (2009)
Sustained effects of nonallele-specific Huntingtin
silencing. Ann Neurol 65(3):276-285
76. Grondin R, Kaytor MD, Ai Y et al (2012) Six-
month partial suppression of Huntingtin is well
tolerated in the adult rhesus striatum. Brain
135(4):1197-1209
77. Grimm D, Streetz KL, Jopling CL et al (2006)
Fatality in mice due to oversaturation of cellu-
lar microRNA/short hairpin RNA pathways.
Nature 441(7092):537-541
78. Olson SD, Kambal A, Pollock K et al (2012)
Examination of mesenchymal stem cell-
mediated RNAi transfer to Huntington's dis-
ease affected neuronal cells for reduction of
huntingtin. Mol Cell Neurosci 49(3):271-281
79. Amarzguioui M, Lundberg P, Cantin E et al
(2006) Rational design and in vitro and in vivo
delivery of Dicer substrate siRNA. Nat Protoc
1(2):508-517
80. Judge AD, Bola G, Lee ACH et al (2006)
Design of noninflammatory synthetic siRNA
mediating potent gene silencing in vivo. Mol
Ther 13(3):494-505
81. Wang YL, Liu W, Wada E et al (2005) Clinico-
pathological rescue of a model mouse of
Huntington's disease by siRNA. Neurosci Res
53(3):241-249
82. DiFiglia M, Sena-Esteves M, Chase K et al
(2007) Therapeutic silencing of mutant hun-
tingtin with siRNA attenuates striatal and corti-
cal neuropathology and behavioral deficits. Proc
Natl Acad Sci U S A 104(43):17204-17209
83. Stanek LM, Sardi SP, Mastis B et al (2014)
Silencing mutant Huntingtin by adeno-
associated virus-mediated RNA interference
ameliorates disease manifestations in the
YAC128 mouse model of Huntington's dis-
ease. Hum Gene Ther 25:461-474
84. Cai X, Hagedorn CH, Cullen BR (2004)
Human microRNAs are processed from
capped, polyadenylated transcripts that can also
function as mRNAs. RNA 10(12):1957-1966
85. Boden D, Pusch O, Silbermann R et al (2004)
Enhanced gene silencing of HIV-1 specific
siRNA using microRNA designed hairpins.
Nucleic Acids Res 32(3):1154-1158
86. Wiznerowicz M, Szulc J, Trono D (2006)
Tuning silence: conditional systems for RNA
interference. Nat Methods 3(9):682-688
87. McBride JL, Boudreau RL, Harper SQ et al
(2008) Artificial miRNAs mitigate shRNA-
mediated toxicity in the brain: implications for
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