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Publications relevant to rAVETM
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Monahan PE, Samulski RJ. Adeno-associated
virus vectors for gene therapy: more pros than cons? Mol Med Today. 2000
6(11):433-40.
Monahan PE, Samulski RJ. AAV vectors: is
clinical success on the horizon? Gene Ther. 2000 7(1):24-30.
Janson CG, McPhee SW, Leone P, Freese A,
During MJ. Viral-based gene transfer to the mammalian CNS for functional
genomic studies. Trends Neurosci. 2001 Dec;24(12):706-12.
rAVE
Janson CG, During MJ. Viral vectors as
part of an integrated functional genomics program. Genomics. 2001
Nov;78(1-2):3-6.
rAVE
Tenenbaum L, Hamdane M, Pouzet M, Avalosse
B, Stathopoulos A, Jurysta F, Rosenbaum C, Hanemann CO, Levivier M, Velu
T. Cellular contaminants of adeno-associated virus vector stocks can
enhance transduction. Gene Ther. 1999 6(6):1045-53.
Lai YK, Rolling F, Baker E, Rakoczy PE.
Kinetics of efficient recombinant adeno-associated virus transduction in
retinal pigment epithelial cells. Exp Cell Res. 2001 267(2):184-92.
Owen R IV, Lewin AP, Peel A, Wang J, Guy
J, Hauswirth WW, Stacpoole PW, Flotte TR. Recombinant adeno-associated
virus vector-based gene transfer for defects in oxidative metabolism.
Hum Gene Ther. 2000 11(15):2067-78.
Seo BB, Wang J, Flotte TR, Yagi T,
Matsuno-Yagi A. Use of the NADH-quinone oxidoreductase (NDI1) gene of
Saccharomyces cerevisiae as a possible cure for complex I defects in
human cells. J Biol Chem. 2000 275(48):37774-8.
Paul D, Qazilbash MH, Song K, Xu H, Sinha
BK, Liu J, Cowan KH. Construction of a recombinant adeno-associated
virus (rAAV) vector expressing murine interleukin-12 (IL-12). Cancer
Gene Ther. 2000 7(2):308-15.
Fu H, Samulski RJ, McCown TJ, Picornell YJ,
Fletcher D, Muenzer J. Neurological correction of lysosomal storage in a
mucopolysaccharidosis IIIB mouse model by adeno-associated
virus-mediated gene delivery. Mol Ther. 2002 5(1):42-9.
Yamano S, Scott DE, Huang LY, Mikolajczyk
M, Pillemer SR, Chiorini JA, Golding B, Baum BJ. Protection from
experimental endotoxemia by a recombinant adeno-associated virus
encoding interleukin 10. J Gene Med. 2001 3(5):450-7.
Ponnazhagan S, Mukherjee P, Wang XS, Qing
K, Kube DM, Mah C, Kurpad C, Yoder MC, Srour EF, Srivastava A. Adeno-associated
virus type 2-mediated transduction in primary human bone marrow-derived
CD34+ hematopoietic progenitor cells: donor variation and correlation of
transgene expression with cellular differentiation. J Virol. 1997
71(11):8262-7.
Nathwani AC, Hanawa H, Vandergriff J,
Kelly P, Vanin EF, Nienhuis AW. Efficient gene transfer into human cord
blood CD34+ cells and the CD34+CD38- subset using highly purified
recombinant adeno-associated viral vector preparations that are free of
helper virus and wild-type AAV. Gene Ther. 2000 7(3):183-95.
Handa A, Muramatsu S, Qiu J, Mizukami H,
Brown KE. Adeno-associated virus (AAV)-3-based vectors transduce
haematopoietic cells not susceptible to transduction with AAV-2-based
vectors. J Gen Virol. 2000 81 Pt 8:2077-84.
Xiao X, Li J, Samulski RJ. Efficient
long-term gene transfer into muscle tissue of immunocompetent mice by
adeno-associated virus vector. J Virol. 1996 70(11):8098-108.
Kessler PD, Podsakoff GM, Chen X,
McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Byrne BJ. Gene
delivery to skeletal muscle results in sustained expression and systemic
delivery of a therapeutic protein. Proc Natl Acad Sci U S A. 1996
93(24):14082-7.
Wang B, Li J, Xiao X. Adeno-associated
virus vector carrying human minidystrophin genes effectively ameliorates
muscular dystrophy in mdx mouse model. Proc Natl Acad Sci U S A. 2000
97(25):13714-9.
Byun J, Heard JM, Huh JE, Park SJ, Jung
EA, Jeong JO, Gwon HC, Kim DK. Efficient expression of the vascular
endothelial growth factor gene in vitro and in vivo, using an adeno-associated
virus vector. J Mol Cell Cardiol. 2001 33(2):295-305.
Rabinowitz JE, Rolling F, Li C, Conrath H,
Xiao W, Xiao X, Samulski RJ. Cross-packaging of a single adeno-associated
virus (AAV) type 2 vector genome into multiple AAV serotypes enables
transduction with broad specificity. J Virol. 2002 76(2):791-801.
Larson PJ, High KA. Gene therapy for
hemophilia B: AAV-mediated transfer of the gene for coagulation factor
IX to human muscle. Adv Exp Med Biol. 2001;489:45-57.
Maeda Y, Ikeda U, Shimpo M, Ueno S,
Ogasawara Y, Urabe M, Kume A, Takizawa T, Saito T, Colosi P, Kurtzman G,
Shimada K, Ozawa K. Efficient gene transfer into cardiac myocytes using
adeno-associated virus (AAV) vectors. J Mol Cell Cardiol. 1998
30(7):1341-8.
Su H, Lu R, Kan YW. Adeno-associated viral
vector-mediated vascular endothelial growth factor gene transfer induces
neovascular formation in ischemic heart. Proc Natl Acad Sci U S A. 2000
97(25):13801-6.
Jung SC, Han IP, Limaye A, Xu R, Gelderman
MP, Zerfas P, Tirumalai K, Murray GJ, During MJ, Brady RO, Qasba P.
Adeno-associated viral vector-mediated gene transfer results in
long-term enzymatic and functional correction in multiple organs of
Fabry mice. Proc Natl Acad Sci U S A. 2001 98(5):2676-81.
rAVE
During MJ, Kaplitt MG, Stern MB, Eidelberg
D. Subthalamic GAD gene transfer in Parkinson disease patients who are
candidates for deep brain stimulation. Hum Gene Ther. 2001
12(12):1589-91.
rAVE
Leone P, Janson CG, Bilaniuk L, Wang Z,
Sorgi F, Huang L, Matalon R, Kaul R, Zeng Z, Freese A, McPhee SW, Mee E,
During MJ, Bilianuk L. Aspartoacylase gene transfer to the mammalian
central nervous system with therapeutic implications for Canavan
disease. Ann Neurol.
2000 48(1):27-38.
rAVE
Mastakov MY, Baer K, Xu R, Fitzsimons H,
During MJ. Combined injection of rAAV with mannitol enhances gene
expression in the rat brain. Mol Ther. 2001 3(2):225-32.
rAVE
Davidson BL, Stein CS, Heth JA, Martins I,
Kotin RM, Derksen TA, Zabner J, Ghodsi A, Chiorini JA. Recombinant adeno-associated
virus type 2, 4, and 5 vectors: transduction of variant cell types and
regions in the mammalian central nervous system. Proc Natl Acad Sci U S
A. 2000 97(7):3428-32.
Halbert CL, Rutledge EA, Allen JM, Russell
DW, Miller AD. Repeat transduction in the mouse lung by using adeno-associated
virus vectors with different serotypes. J Virol. 2000 74(3):1524-32.
Zabner J, Seiler M, Walters R, Kotin RM,
Fulgeras W, Davidson BL, Chiorini JA. Adeno-associated virus type 5
(AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and
facilitates gene transfer. J Virol. 2000 74(8):3852-8.
Lipshutz GS, Gruber CA, Cao Y, Hardy J,
Contag CH, Gaensler KM. In utero delivery of adeno-associated viral
vectors: intraperitoneal gene transfer produces long-term expression.
Mol Ther. 2001 3(3):284-92.
Flotte TR, Laube BL. Gene therapy in
cystic fibrosis. Chest. 2001 120(3 Suppl):124S-131S.
Aitken ML, Moss RB, Waltz DA, Dovey ME,
Tonelli MR, McNamara SC, Gibson RL, Ramsey BW, Carter BJ, Reynolds TC. A
phase I study of aerosolized administration of tgAAVCF to cystic
fibrosis subjects with mild lung disease. Hum Gene Ther. 2001
12(15):1907-16.
Sarra GM, Stephens C, de Alwis M,
Bainbridge JW, Smith AJ, Thrasher AJ, Ali RR. Gene replacement therapy
in the retinal degeneration slow (rds) mouse: the effect on retinal
degeneration following partial transduction of the retina. Hum Mol
Genet. 2001 10(21):2353-61.
McGee Sanftner LH, Rendahl KG, Quiroz D,
Coyne M, Ladner M, Manning WC, Flannery JG. Recombinant AAV-mediated
delivery of a tet-inducible reporter gene to the rat retina. Mol Ther.
2001 3(5 Pt 1):688-96.
Jomary C, Grist J, Milbrandt J, Neal MJ,
Jones SE. Epitope-tagged recombinant AAV vectors for expressing
neurturin and its receptor in retinal cells. Mol Vis. 2001 7:36-41.
During MJ, Xu R, Young D, Kaplitt MG,
Sherwin RS, Leone P. Peroral gene therapy of lactose intolerance using
an adeno-associated virus vector. Nat Med. 1998 4(10):1131-5.
rAVE
During MJ, Symes CW, Lawlor PA, Lin J,
Dunning J, Fitzsimons HL, Poulsen D, Leone P, Xu R, Dicker BL, Lipski J,
Young D. An oral vaccine against NMDAR1 with efficacy in experimental
stroke and epilepsy. Science. 2000 287(5457):1453-60.
rAVE
Schwaab R, Oldenburg J. Gene therapy of
hemophilia. Semin Thromb Hemost. 2001 27(4):417-24.
Duan D, Yan Z, Yue Y, Ding W, Engelhardt
JF. Enhancement of muscle gene delivery with pseudotyped adeno-associated
virus type 5 correlates with myoblast differentiation. J Virol. 2001
75(16):7662-71.
Sanchez-Pernaute R, Harvey-White J,
Cunningham J, Bankiewicz KS. Functional effect of adeno-associated virus
mediated gene transfer of aromatic L-amino acid decarboxylase into the
striatum of 6-OHDA-lesioned rats. Mol Ther. 2001 4(4):324-30.
Kirik D, Rosenblad C, Bjorklund A, Mandel
RJ. Long-term rAAV-mediated gene transfer of GDNF in the rat Parkinson's
model: intrastriatal but not intranigral transduction promotes
functional regeneration in the lesioned nigrostriatal system. J Neurosci.
2000 20(12):4686-700.
Leone P, Janson CG, McPhee SJ, During MJ.
Global CNS gene transfer for a childhood neurogenetic enzyme deficiency:
Canavan disease. Curr Opin Mol Ther. 1999 1(4):487-92.
rAVE
Cottard V, Mulleman D, Bouille P, Mezzina
M, Boissier MC, Bessis N. Adeno-associated virus-mediated delivery of
IL-4 prevents collagen-induced arthritis. Gene Ther. 2000 7(22):1930-9.
Akagi K, Sandig V, Vooijs M, Van der Valk
M, Giovannini M, Strauss M, Berns A. Cre-mediated somatic site-specific
recombination in mice. Nucleic Acids Res. 1997 25(9):1766-73.
Meuwissen R, Linn SC, van der Valk M, Mooi
WJ, Berns A. Mouse model for lung tumorigenesis through Cre/lox
controlled sporadic activation of the K-Ras oncogene. Oncogene. 2001
20(45):6551-8.
South S, Bogulavsky J, Franklin S, Martin
D, Vissel B, Sailer A, Malkmus S, Masuyama T, Horner P, Kaspar B, Gage
F, Tonegawa S, Yaksh T, Heinemann S, Inturissi CE. A conditional
knockout (KO) of the NMDAR1 subunit in spinal cord dorsal horn
attentuates hyperalgesia. Soc. Neurosci Abstr. 2001, 53.4
Bengoechea TG, Lin W, Kaspar B, Harada N,
Domingez B, Gage FH, Lee KL. A conditional mouse mutation system for
spatiotemporal removal of the neurotrophin receptor P75 to study its
role in the adult basal forebrain. Soc. Neurosci. Abstr. 2001, 28.3.
Greene RW, Lee JS, Gray J, Beaudet M,
Estabrooke IV, Thompson MA, Saper CB, Scammell TE. Anatomically specific
deletion of the adenosine A1 receptor using an adeno-associated viral
vector. Soc. Neurosci. Abstr. 2001, 523.5.
Klein RL, McNamara RK, King MA, Lenox RH,
Muzyczka N, Meyer EM. Generation of aberrant sprouting in the adult rat
brain by GAP-43 somatic gene transfer. Brain Res. 1999 832(1-2):136-44.
Kimura B, Mohuczy D, Tang X, Phillips MI.
Attenuation of hypertension and heart hypertrophy by adeno-associated
virus delivering angiotensinogen antisense. Hypertension. 2001 37(2 Part
2):376-80.
Phillips MI. Gene therapy for
hypertension: sense and antisense strategies. Expert Opin Biol Ther.
2001 1(4):655-62.
Senut MC, Suhr ST, Kaspar B, Gage FH.
Intraneuronal aggregate formation and cell death after viral expression
of expanded polyglutamine tracts in the adult rat brain. J Neurosci.
2000 20(1):219-29.
Rossi FM, Guicherit OM, Spicher A,
Kringstein AM, Fatyol K, Blakely BT, Blau HM. Tetracycline-regulatable
factors with distinct dimerization domains allow reversible growth
inhibition by p16. Nat Genet. 1998 20(4):389-93.
Haberman RP, McCown TJ, Samulski RJ.
Inducible long-term gene expression in brain with adeno-associated virus
gene transfer.Gene Ther. 1998 5(12):1604-11.
Manning WC, Paliard X, Zhou S, Pat Bland
M, Lee AY, Hong K, Walker CM, Escobedo JA, Dwarki V. Genetic
immunization with adeno-associated virus vectors expressing herpes
simplex virus type 2 glycoproteins B and D. J Virol. 1997 71(10):7960-2.
Brockstedt DG, Podsakoff GM, Fong L,
Kurtzman G, Mueller-Ruchholtz W, Engleman EG. Induction of immunity to
antigens expressed by recombinant adeno-associated virus depends on the
route of administration. Clin Immunol. 1999 92(1):67-75.
Xin KQ, Urabe M, Yang J, Nomiyama K,
Mizukami H, Hamajima K, Nomiyama H, Saito T, Imai M, Monahan J, Okuda K,
Ozawa K, Okuda K. A novel recombinant adeno-associated virus vaccine
induces a long-term humoral immune response to human immunodeficiency
virus. Hum Gene Ther. 2001 12(9):1047-61.
Chamberlin NL, Du B, de Lacalle S, Saper
CB. Recombinant adeno-associated virus vector: use for transgene
expression and anterograde tract tracing in the CNS. Brain Res. 1998
793(1-2):169-75.
Zolotukhin S, Byrne BJ, Mason E,
Zolotukhin I, Potter M, Chesnut K, Summerford C, Samulski RJ, Muzyczka
N. Recombinant adeno-associated virus purification using novel methods
improves infectious titer and yield. Gene Ther. 1999 6(6):973-85.
Clark KR, Liu X, McGrath JP, Johnson PR.
Highly purified recombinant adeno-associated virus vectors are
biologically active and free of detectable helper and wild-type viruses.
Hum Gene Ther. 1999 10(6):1031-9.
Summerford C, Samulski RJ.
Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated
virus type 2 virions. J Virol. 1998 72(2):1438-45.
Qing K, Mah C, Hansen J, Zhou S, Dwarki V,
Srivastava A.Human fibroblast growth factor receptor 1 is a co-receptor
for infection by adeno-associated virus 2. Nat Med. 1999 5(1):71-7.
Rutledge EA, Halbert CL, Russell DW.
Infectious clones and vectors derived from adeno associated virus (AAV)
serotypes other than AAV type 2. J Virol. 1998 72(1):309-19.
Rabinowitz JE, Rolling F, Li C, Conrath H,
Xiao W, Xiao X, Samulski RJ. Cross-packaging of a single adeno-associated
virus (AAV) type 2 vector genome into multiple AAV serotypes enables
transduction with broad specificity. J Virol. 2002 Jan;76(2):791-801.
Xu L, Daly T, Gao C, Flotte TR, Song S,
Byrne BJ, Sands MS, Parker Ponder K. CMV-beta-actin promoter directs
higher expression from an adeno-associated viral vector in the liver
than the cytomegalovirus or elongation factor 1 alpha promoter and
results in therapeutic levels of human factor X in mice. Hum Gene Ther.
2001 12(5):563-73.
Donello JE, Loeb JE, Hope TJ. Woodchuck
hepatitis virus contains a tripartite posttranscriptional regulatory
element. J Virol. 1998 72(6):5085-92.
Paterna JC, Moccetti T, Mura A, Feldon J,
Bueler H. Influence of promoter and WHV post-transcriptional regulatory
element on AAV-mediated transgene expression in the rat brain. Gene Ther.
2000 7(15):1304-11.
Auten J, Agarwal M, Chen J, Sutton R,
Plavec I. Effect of scaffold attachment region on transgene expression
in retrovirus vector-transduced primary T cells and macrophages. Hum
Gene Ther. 1999 10(8):1389-99.
Agarwal M, Austin TW, Morel F, Chen J,
Bohnlein E, Plavec I.Scaffold attachment region-mediated enhancement of
retroviral vector expression in primary T cells. J Virol. 1998
72(5):3720-8.
Alexander IE, Russell DW, Miller AD.
Transfer of contaminants in adeno-associated virus vector stocks can
mimic transduction and lead to artifactual results. Hum Gene Ther. 1997
8(16):1911-20.
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