Biology Reference
In-Depth Information
56. Foust KD et al (2010) Rescue of the spinal
muscular atrophy phenotype in a mouse model
by early postnatal delivery of SMN. Nat
Biotechnol 28(3):271-274
57. Spampanato C et al (2011) Effi cacy of a com-
bined intracerebral and systemic gene delivery
approach for the treatment of a severe lysosomal
storage disorder. Mol Ther 19(5):860-869
58. Gray SJ et al (2011) Preclinical differences of
intravascular AAV9 delivery to neurons and
glia: a comparative study of adult mice and non-
human primates. Mol Ther 19(6):1058-1069
59. Bevan AK et al (2011) Systemic gene delivery
in large species for targeting spinal cord, brain,
and peripheral tissues for pediatric disorders.
Mol Ther 19(11):1971-1980
60. Samaranch L et al (2012) Adeno-associated
virus serotype 9 transduction in the central
nervous system of nonhuman primates. Hum
Gene Ther 23(4):382-389
61. Dehay B, Dalkara D, Dovero S, Li Q, Bezard
E (2012) Systemic scAAV9 variant mediates
brain transduction in newborn rhesus
macaques. Sci Rep 2:253
62. Mattar CN et al (2013) Systemic delivery of
scAAV9 in fetal macaques facilitates neuronal
transduction of the central and peripheral ner-
vous systems. Gene Ther 20(1):69-83
63. Federici T et al (2012) Robust spinal motor
neuron transduction following intrathecal
delivery of AAV9 in pigs. Gene Ther
19(8):852-859
64. Xie J et al (2011) MicroRNA-regulated, sys-
temically delivered rAAV9: a step closer to
CNS-restricted transgene expression. Mol
Ther 19(3):526-535
65. Asokan A, Schaffer DV, Samulski RJ (2012)
The AAV vector toolkit: poised at the clinical
crossroads. Mol Ther 20(4):699-708
66. Gray SJ et al (2010) Directed evolution of a
novel adeno-associated virus (AAV) vector that
crosses the seizure-compromised blood-brain
barrier (BBB). Mol Ther 18(3):570-578
67. Asokan A et al (2010) Reengineering a recep-
tor footprint of adeno-associated virus enables
selective and systemic gene transfer to muscle.
Nat Biotechnol 28(1):79-82
68. Gray SJ, Nagabhushan Kalburgi S, McCown
TJ, Jude Samulski R (2013) Global CNS gene
delivery and evasion of anti-AAV-neutralizing
antibodies by intrathecal AAV administration
in
(AAV1) in neonatal mice results in comple-
mentary patterns of neuronal transduction to
AAV2 and total long-term correction of stor-
age lesions in the brains of beta-glucuronidase-
defi cient mice. J Virol 77(12):7034-7040
71. Regev L, Ezrielev E, Gershon E, Gil S, Chen A
(2010) Genetic approach for intracerebroven-
tricular delivery. Proc Natl Acad Sci USA
107(9):4424-4429
72. Lam MF, Thomas MG, Lind CR (2011)
Neurosurgical convection-enhanced delivery
of treatments for Parkinson's disease. J Clin
Neurosci 18(9):1163-1167
73. Bobo RH et al (1994) Convection-enhanced
delivery of macromolecules in the brain. Proc
Natl Acad Sci USA 91(6):2076-2080
74. Cunningham J et al (2008) Biodistribution of
adeno-associated virus type-2 in nonhuman
primates after convection-enhanced delivery to
brain. Mol Ther 16(7):1267-1275
75. Fiandaca MS, Forsayeth JR, Dickinson PJ,
Bankiewicz KS (2008) Image-guided
convection-enhanced delivery platform in the
treatment
of
neurological
diseases.
Neurotherapeutics 5(1):123-127
76. Salegio EA, Samaranch L, Kells AP, Forsayeth
J, Bankiewicz K (2012) Guided delivery of
adeno-associated viral vectors into the primate
brain. Adv Drug Deliv Rev 64(7):598-604
77. Chen ZJ et al (2004) A realistic brain tissue
phantom for intraparenchymal infusion stud-
ies. J Neurosurg 101(2):314-322
78. Fatouros PP et al (2006) In vitro and in vivo
imaging studies of a new endohedral
metallofullerene
nanoparticle.
Radiology
240(3):756-764
79. Krauze MT et al (2005) Refl ux-free cannula
for convection-enhanced high-speed delivery
of therapeutic agents. J Neurosurg 103(5):
923-929
80. Rosenbluth KH et al (2011) Design of an in-
dwelling cannula for convection-enhanced
delivery. J Neurosci Methods 196(1):118-123
81. Sanftner LM et al (2005) AAV2-mediated
gene delivery to monkey putamen: evaluation
of an infusion device and delivery parameters.
Exp Neurol 194(2):476-483
82. Saito R et al (2005) Gadolinium-loaded lipo-
somes allow for real-time magnetic resonance
imaging of convection-enhanced delivery in the
primate brain. Exp Neurol 196(2):381-389
83. Yin D et al (2011) Optimal region of the puta-
men for image-guided convection-enhanced
delivery of therapeutics in human and non-
human primates. Neuroimage 54(Suppl
1):S196-S203
84. Eberling JL et al (2008) Results from a phase I
safety trial of hAADC gene therapy for Parkinson
disease. Neurology 70(21):1980-1983
non-human
primates.
Gene
Ther
20(4):450-459
69. Bielicki J, McIntyre C, Anson DS (2010)
Comparison of ventricular and intravenous
lentiviral-mediated gene therapy for murine
MPS VII. Mol Genet Metab 101(4):370-382
70. Passini MA et al (2003) Intraventricular brain
injection of adeno-associated virus type 1