-
2
-
-
2342666229
-
Enzyme replacement therapy for mucopolysaccharidosis I: A randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-l-iduronidase (laronidase)
-
J.E. Wraith, L.A. Clarke, M. Beck, E.H. Kolodny, G.M. Pastores, J. Muenzer, D.M. Rapoport, K.I. Berger, S.J. Swiedler, E.D. Kakkis, T. Braakman, E. Chadbourne, K. Walton-Bowen, and G.F. Cox Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-l-iduronidase (laronidase) J. Pediatr. 144 2004 581 588
-
(2004)
J. Pediatr.
, vol.144
, pp. 581-588
-
-
Wraith, J.E.1
Clarke, L.A.2
Beck, M.3
Kolodny, E.H.4
Pastores, G.M.5
Muenzer, J.6
Rapoport, D.M.7
Berger, K.I.8
Swiedler, S.J.9
Kakkis, E.D.10
Braakman, T.11
Chadbourne, E.12
Walton-Bowen, K.13
Cox, G.F.14
-
3
-
-
0035718871
-
Enzyme replacement therapy in feline mucopolysaccharidosis I
-
E.D. Kakkis, E. Schuchman, X. He, Q. Wan, S. Kania, S. Wiemelt, C.W. Hasson, T. O'Malley, M.A. Weil, G.A. Aguirre, D.E. Brown, and M.E. Haskins Enzyme replacement therapy in feline mucopolysaccharidosis I Mol. Genet. Metab. 72 2001 199 208
-
(2001)
Mol. Genet. Metab.
, vol.72
, pp. 199-208
-
-
Kakkis, E.D.1
Schuchman, E.2
He, X.3
Wan, Q.4
Kania, S.5
Wiemelt, S.6
Hasson, C.W.7
O'Malley, T.8
Weil, M.A.9
Aguirre, G.A.10
Brown, D.E.11
Haskins, M.E.12
-
4
-
-
0028568013
-
Enzyme replacement in a canine model of Hurler syndrome
-
R.M. Shull, E.D. Kakkis, M.F. McEntee, S.A. Kania, A.J. Jonas, and E.F. Neufeld Enzyme replacement in a canine model of Hurler syndrome Proc. Natl. Acad. Sci. USA 91 1994 12937 12941
-
(1994)
Proc. Natl. Acad. Sci. USA
, vol.91
, pp. 12937-12941
-
-
Shull, R.M.1
Kakkis, E.D.2
McEntee, M.F.3
Kania, S.A.4
Jonas, A.J.5
Neufeld, E.F.6
-
5
-
-
0035905889
-
Enzyme replacement therapy in mucopolysaccharidosis I
-
E.D. Kakkis, J. Muenzer, G.E. Tiller, L. Waber, J. Belmont, M. Passage, B. Izykowski, J. Phillips, R. Doroshow, I. Walot, R. Hoft, and E.F. Neufeld Enzyme replacement therapy in mucopolysaccharidosis I New Engl. J. Med. 344 2001 182 188
-
(2001)
New Engl. J. Med.
, vol.344
, pp. 182-188
-
-
Kakkis, E.D.1
Muenzer, J.2
Tiller, G.E.3
Waber, L.4
Belmont, J.5
Passage, M.6
Izykowski, B.7
Phillips, J.8
Doroshow, R.9
Walot, I.10
Hoft, R.11
Neufeld, E.F.12
-
6
-
-
0032904141
-
Delivery, distribution, and neuronal uptake of exogenous mannose-terminal glucocerebrosidase in the intact rat brain
-
G.C. Zirzow, O.A. Sanchez, G.J. Murray, R.O. Brady, and E.H. Oldfield Delivery, distribution, and neuronal uptake of exogenous mannose-terminal glucocerebrosidase in the intact rat brain Neurochem. Res. 24 1999 301 305
-
(1999)
Neurochem. Res.
, vol.24
, pp. 301-305
-
-
Zirzow, G.C.1
Sanchez, O.A.2
Murray, G.J.3
Brady, R.O.4
Oldfield, E.H.5
-
7
-
-
0035120218
-
Heparin coinfusion during convection-enhanced delivery (CED) increases the distribution of the glial-derived neurotropic factor (GDNF) ligand family in rat striatum and enhances the pharmacological activity of neurturin
-
J.F. Hamilton, P.F. Morrison, M.Y. Chen, J. Harvey-White, R.S. Pernaute, H. Phillips, E. Oldfield, and K.S. Bankiewicz Heparin coinfusion during convection-enhanced delivery (CED) increases the distribution of the glial-derived neurotropic factor (GDNF) ligand family in rat striatum and enhances the pharmacological activity of neurturin Exp. Neurol. 168 2001 155 161
-
(2001)
Exp. Neurol.
, vol.168
, pp. 155-161
-
-
Hamilton, J.F.1
Morrison, P.F.2
Chen, M.Y.3
Harvey-White, J.4
Pernaute, R.S.5
Phillips, H.6
Oldfield, E.7
Bankiewicz, K.S.8
-
8
-
-
0037079071
-
Drug and gene delivery to the brain: The vascular route
-
W.M. Pardridge Drug and gene delivery to the brain: the vascular route Neuron 36 2002 555 558
-
(2002)
Neuron
, vol.36
, pp. 555-558
-
-
Pardridge, W.M.1
-
9
-
-
4544321276
-
Lipoprotein receptor binding, cellular uptake, and lysosomal delivery of fusions between the receptor-associated protein (RAP) and α-l-iduronidase or acid α-glucosidase
-
W.S. Prince, L.M. McCormick, D.J. Wendt, P.A. Fitzpatrick, K.L. Schwartz, A.I. Aguilera, V. Koppaka, T.M. Christianson, M.C. Vellard, N. Pavloff, J.F. Lemontt, M. Qin, C.M. Starr, G. Bu, and T.C. Zankel Lipoprotein receptor binding, cellular uptake, and lysosomal delivery of fusions between the receptor-associated protein (RAP) and α-l-iduronidase or acid α-glucosidase J. Biol. Chem. 279 2004 35037 35046
-
(2004)
J. Biol. Chem.
, vol.279
, pp. 35037-35046
-
-
Prince, W.S.1
McCormick, L.M.2
Wendt, D.J.3
Fitzpatrick, P.A.4
Schwartz, K.L.5
Aguilera, A.I.6
Koppaka, V.7
Christianson, T.M.8
Vellard, M.C.9
Pavloff, N.10
Lemontt, J.F.11
Qin, M.12
Starr, C.M.13
Bu, G.14
Zankel, T.C.15
-
10
-
-
18744415035
-
High transcytosis of melanotransferrin (P97) across the blood-brain barrier
-
M. Demeule, J. Poirier, J. Jodoin, Y. Bertrand, R.R. Desrosiers, C. Dagenais, T. Nguyen, J. Lanthier, R. Gabathuler, M. Kennard, W.A. Jefferies, D. Karkan, S. Tsai, L. Fenart, R. Cecchelli, and R. Béliveau High transcytosis of melanotransferrin (P97) across the blood-brain barrier J. Neurochem. 83 2002 924 933
-
(2002)
J. Neurochem.
, vol.83
, pp. 924-933
-
-
Demeule, M.1
Poirier, J.2
Jodoin, J.3
Bertrand, Y.4
Desrosiers, R.R.5
Dagenais, C.6
Nguyen, T.7
Lanthier, J.8
Gabathuler, R.9
Kennard, M.10
Jefferies, W.A.11
Karkan, D.12
Tsai, S.13
Fenart, L.14
Cecchelli, R.15
Béliveau, R.16
-
11
-
-
0028826147
-
Delivery of herpesvirus and adenovirus to nude rat intracerebral tumors after osmotic blood-brain barrier disruption
-
G. Nilaver, L.L. Muldoon, R.A. Kroll, M.A. Pagel, X.O. Breakefield, B.L. Davidson, and E.A. Neuwelt Delivery of herpesvirus and adenovirus to nude rat intracerebral tumors after osmotic blood-brain barrier disruption Proc. Natl. Acad. Sci. USA 92 1995 9829 9833
-
(1995)
Proc. Natl. Acad. Sci. USA
, vol.92
, pp. 9829-9833
-
-
Nilaver, G.1
Muldoon, L.L.2
Kroll, R.A.3
Pagel, M.A.4
Breakefield, X.O.5
Davidson, B.L.6
Neuwelt, E.A.7
-
12
-
-
2942553230
-
VEGF disrupts the neonatal blood-brain barrier and increases life span after non-ablative BMT in a murine model of congenital neurodegeneration caused by a lysosomal enzyme deficiency
-
P.P. Young, C.R. Fantz, and M.S. Sands VEGF disrupts the neonatal blood-brain barrier and increases life span after non-ablative BMT in a murine model of congenital neurodegeneration caused by a lysosomal enzyme deficiency Exp. Neurol. 188 2004 104 114
-
(2004)
Exp. Neurol.
, vol.188
, pp. 104-114
-
-
Young, P.P.1
Fantz, C.R.2
Sands, M.S.3
-
13
-
-
23444437213
-
Convection-enhanced delivery of macromolecules in the brain
-
R.H. Bobo, D.W. Laske, A. Akbasak, P.F. Morrison, R.L. Dedrick, and E.H. Oldfield Convection-enhanced delivery of macromolecules in the brain Proc. Natl. Acad. Sci. USA 91 1994 2076 2080
-
(1994)
Proc. Natl. Acad. Sci. USA
, vol.91
, pp. 2076-2080
-
-
Bobo, R.H.1
Laske, D.W.2
Akbasak, A.3
Morrison, P.F.4
Dedrick, R.L.5
Oldfield, E.H.6
-
14
-
-
0031451777
-
Tumor regression with regional distribution of the targeted toxin TF-CRM107 in patients with malignant brain tumors
-
D.W. Laske, R.J. Youle, and E.H. Oldfield Tumor regression with regional distribution of the targeted toxin TF-CRM107 in patients with malignant brain tumors Nat. Med. 3 1997 1362 1368
-
(1997)
Nat. Med.
, vol.3
, pp. 1362-1368
-
-
Laske, D.W.1
Youle, R.J.2
Oldfield, E.H.3
-
15
-
-
10744219583
-
Progress report of a Phase I study of the intracerebral microinfusion of a recombinant chimeric protein composed of transforming growth factor (TGF)-α and a mutated form of the Pseudomonas exotoxin termed PE-38 (TP-38) for the treatment of malignant brain tumors
-
J.H. Sampson, G. Akabani, G.E. Archer, D.D. Bigner, M.S. Berger, A.H. Friedman, H.S. Friedman, J.E. Herndon II, S. Kunwar, S. Marcus, R.E. McLendon, A. Paolino, K. Penne, J. Provenzale, J. Quinn, D.A. Reardon, J. Rich, T. Stenzel, S. Tourt-Uhlig, C. Wikstrand, T. Wong, R. Williams, F. Yuan, M.R. Zalutsky, and I. Pastan Progress report of a Phase I study of the intracerebral microinfusion of a recombinant chimeric protein composed of transforming growth factor (TGF)-α and a mutated form of the Pseudomonas exotoxin termed PE-38 (TP-38) for the treatment of malignant brain tumors J. Neuro-Oncol. 65 2003 27 35
-
(2003)
J. Neuro-Oncol.
, vol.65
, pp. 27-35
-
-
Sampson, J.H.1
Akabani, G.2
Archer, G.E.3
Bigner, D.D.4
Berger, M.S.5
Friedman, A.H.6
Friedman, H.S.7
Herndon II, J.E.8
Kunwar, S.9
Marcus, S.10
McLendon, R.E.11
Paolino, A.12
Penne, K.13
Provenzale, J.14
Quinn, J.15
Reardon, D.A.16
Rich, J.17
Stenzel, T.18
Tourt-Uhlig, S.19
Wikstrand, C.20
Wong, T.21
Williams, R.22
Yuan, F.23
Zalutsky, M.R.24
Pastan, I.25
more..
-
16
-
-
0344976256
-
Enzyme replacement therapy in acute neuronopathic Gaucher's patient
-
Milano, Italy, May 27
-
B. Bembi, M. Zanatta, R. Boscolo, M. Bottega, V. Forleo, M. Carrozi, Enzyme replacement therapy in acute neuronopathic Gaucher's patient, Abstr. VI Int. Congress on Inborn Errors of Metabolism, Milano, Italy, May 27, 1994.
-
(1994)
Abstr. VI Int. Congress on Inborn Errors of Metabolism
-
-
Bembi, B.1
Zanatta, M.2
Boscolo, R.3
Bottega, M.4
Forleo, V.5
Carrozi, M.6
-
17
-
-
0029001712
-
Convection-enhanced distribution of large molecules in gray matter during interstitial drug infusion
-
D.M. Lieberman, D.W. Laske, P.F. Morrison, K.S. Bankiewicz, and E.H. Oldfield Convection-enhanced distribution of large molecules in gray matter during interstitial drug infusion J. Neurosurg. 82 1995 1021 1029
-
(1995)
J. Neurosurg.
, vol.82
, pp. 1021-1029
-
-
Lieberman, D.M.1
Laske, D.W.2
Morrison, P.F.3
Bankiewicz, K.S.4
Oldfield, E.H.5
-
18
-
-
0038249170
-
Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease
-
S.S. Gill, N.K. Patel, G.R. Hotton, K. O'Sullivan, R. McCarter, M. Bunnage, D.J. Brooks, C.N. Svendsen, and P. Heywood Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease Nat. Med. 9 2003 589 595
-
(2003)
Nat. Med.
, vol.9
, pp. 589-595
-
-
Gill, S.S.1
Patel, N.K.2
Hotton, G.R.3
O'Sullivan, K.4
McCarter, R.5
Bunnage, M.6
Brooks, D.J.7
Svendsen, C.N.8
Heywood, P.9
-
19
-
-
18344368012
-
Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors
-
A.I. Brooks, C.S. Stein, S.M. Hughes, J. Heth, P.M. McCray Jr., S.L. Sauter, J.C. Johnston, D.A. Cory-Slechta, H.J. Federoff, and B.L. Davidson Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors Proc. Natl. Acad. Sci. USA 99 2002 6216 6221
-
(2002)
Proc. Natl. Acad. Sci. USA
, vol.99
, pp. 6216-6221
-
-
Brooks, A.I.1
Stein, C.S.2
Hughes, S.M.3
Heth, J.4
McCray Jr., P.M.5
Sauter, S.L.6
Johnston, J.C.7
Cory-Slechta, D.A.8
Federoff, H.J.9
Davidson, B.L.10
-
20
-
-
0034986419
-
Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII
-
W.A. Frisella, L.H. O'Conner, C.A. Vogler, M. Roberts, S. Walkley, B. Levy, T.M. Daly, and M.S. Sands Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII Mol. Ther. 3 2001 351 358
-
(2001)
Mol. Ther.
, vol.3
, pp. 351-358
-
-
Frisella, W.A.1
O'Conner, L.H.2
Vogler, C.A.3
Roberts, M.4
Walkley, S.5
Levy, B.6
Daly, T.M.7
Sands, M.S.8
-
21
-
-
0035099437
-
In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: Correction of neuropathology and protection against learning impairments in affected mice
-
A. Consiglio, A. Quattrini, S. Martino, J.C. Bensadoun, D. Dolcetta, A. Trojani, G. Benaglia, S. Marchesini, V. Cestari, A. Oliverio, C. Bordignon, and L. Naldini In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice Nat. Med. 7 2001 310 316
-
(2001)
Nat. Med.
, vol.7
, pp. 310-316
-
-
Consiglio, A.1
Quattrini, A.2
Martino, S.3
Bensadoun, J.C.4
Dolcetta, D.5
Trojani, A.6
Benaglia, G.7
Marchesini, S.8
Cestari, V.9
Oliverio, A.10
Bordignon, C.11
Naldini, L.12
-
22
-
-
2942596259
-
Treatment of lysosomal storage disorders: Cell therapy and gene therapy
-
Y. Eto, J.-S. Shen, X.-L. Meng, and T. Ohashi Treatment of lysosomal storage disorders: cell therapy and gene therapy J. Inherit. Metab. Dis. 27 2004 411 415
-
(2004)
J. Inherit. Metab. Dis.
, vol.27
, pp. 411-415
-
-
Eto, Y.1
Shen, J.-S.2
Meng, X.-L.3
Ohashi, T.4
-
23
-
-
5444243992
-
Development of efficient plasmid DNA transfer into adult rat central nervous system using microbubble-enhanced ultrasound
-
M. Shimamura, N. Sato, Y. Taniyama, S. Yamamoto, M. Endoh, H. Kurinami, M. Aoki, T. Ogihara, Y. Kaneda, and R. Morishita Development of efficient plasmid DNA transfer into adult rat central nervous system using microbubble-enhanced ultrasound Gene Ther. 11 2004 1532 1539
-
(2004)
Gene Ther.
, vol.11
, pp. 1532-1539
-
-
Shimamura, M.1
Sato, N.2
Taniyama, Y.3
Yamamoto, S.4
Endoh, M.5
Kurinami, H.6
Aoki, M.7
Ogihara, T.8
Kaneda, Y.9
Morishita, R.10
-
24
-
-
3042836949
-
Prevention of neuropathology in the mouse model of Hurler syndrome
-
N. Desmaris, L. Verot, J.P. Puech, C. Caillaud, M.T. Vanier, and J.M. Heard Prevention of neuropathology in the mouse model of Hurler syndrome Ann. Neurol. 56 2004 68 76
-
(2004)
Ann. Neurol.
, vol.56
, pp. 68-76
-
-
Desmaris, N.1
Verot, L.2
Puech, J.P.3
Caillaud, C.4
Vanier, M.T.5
Heard, J.M.6
-
25
-
-
19644400077
-
Viral vectors as tools to model and treat neurodegenerative disorders
-
N. Déglon, and P. Hantraye Viral vectors as tools to model and treat neurodegenerative disorders J. Gene Med. 7 2005 530 539
-
(2005)
J. Gene Med.
, vol.7
, pp. 530-539
-
-
Déglon, N.1
Hantraye, P.2
-
26
-
-
0142084745
-
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
-
S. Hacein-Bey-Abina, C. Von Kalle, M. Schmidt, M.P. McCormack, N. Wulffraat, P. Leboulch, A. Lim, C.S. Osborne, R. Pawliuk, E. Morillon, R. Sorenson, A. Forster, P. Fraser, J.I. Cohen, G. deSaint Basile, I. Alexander, U. Wintergerst, T. Frebourg, A. Aurias, D. Stoppa-Lyonnet, S. Romana, I. Radford-Weiss, F. Gross, F. Valensi, E. Delabesse, E. Macintyre, F. Sigaux, J. Soulier, L.E. Leiva, M. Wissler, C. Prinz, T.H. Rabbitts, F. Le Deist, A. Fischer, and M. Cavazzana-Calvo LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 Science 302 2003 415 419
-
(2003)
Science
, vol.302
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
McCormack, M.P.4
Wulffraat, N.5
Leboulch, P.6
Lim, A.7
Osborne, C.S.8
Pawliuk, R.9
Morillon, E.10
Sorenson, R.11
Forster, A.12
Fraser, P.13
Cohen, J.I.14
Desaint Basile, G.15
Alexander, I.16
Wintergerst, U.17
Frebourg, T.18
Aurias, A.19
Stoppa-Lyonnet, D.20
Romana, S.21
Radford-Weiss, I.22
Gross, F.23
Valensi, F.24
Delabesse, E.25
MacIntyre, E.26
Sigaux, F.27
Soulier, J.28
Leiva, L.E.29
Wissler, M.30
Prinz, C.31
Rabbitts, T.H.32
Le Deist, F.33
Fischer, A.34
Cavazzana-Calvo, M.35
more..
-
27
-
-
0142089747
-
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
-
S.E. Raper, N. Chirmule, F.S. Lee, N.A. Wivel, A. Bagg, G. Gao, J.M. Wilson, and M.L. Batshaw Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer Mol. Genet. Metab. 80 2003 148 158
-
(2003)
Mol. Genet. Metab.
, vol.80
, pp. 148-158
-
-
Raper, S.E.1
Chirmule, N.2
Lee, F.S.3
Wivel, N.A.4
Bagg, A.5
Gao, G.6
Wilson, J.M.7
Batshaw, M.L.8
-
28
-
-
11844303500
-
Recent developments in gene transfer: Risk and ethics
-
J. Kimmelman Recent developments in gene transfer: risk and ethics Br. Med. J. 330 2005 79 82
-
(2005)
Br. Med. J.
, vol.330
, pp. 79-82
-
-
Kimmelman, J.1
-
29
-
-
0018402872
-
Modification of the blood-brain barrier: Increased concentration and fate of enzymes entering the brain
-
J.A. Barranger, S.I. Rapoport, W.R. Fredericks, P.G. Pentchev, K.D. MacDermot, J.K. Steusing, and R.O. Brady Modification of the blood-brain barrier: increased concentration and fate of enzymes entering the brain Proc. Natl. Acad. Sci. USA 76 1979 481 485
-
(1979)
Proc. Natl. Acad. Sci. USA
, vol.76
, pp. 481-485
-
-
Barranger, J.A.1
Rapoport, S.I.2
Fredericks, W.R.3
Pentchev, P.G.4
MacDermot, K.D.5
Steusing, J.K.6
Brady, R.O.7
-
30
-
-
0021281977
-
Delivery of active hexosaminidase across the blood-brain barrier in rats
-
E.A. Neuwelt, J.A. Barranger, M.A. Pagel, J.M. Quirk, R.O. Brady, and E.P. Frenkel Delivery of active hexosaminidase across the blood-brain barrier in rats Neurology 34 1984 1012 1019
-
(1984)
Neurology
, vol.34
, pp. 1012-1019
-
-
Neuwelt, E.A.1
Barranger, J.A.2
Pagel, M.A.3
Quirk, J.M.4
Brady, R.O.5
Frenkel, E.P.6
-
31
-
-
8044258359
-
Uptake of alpha-(l)-iduronidase produced by retrovirally transduced fibroblasts into neuronal and glial cells in vitro
-
K. Stewart, O.A. Brown, A.E. Morelli, L.J. Fairbairn, L.S. Lashford, A. Cooper, C.E. Hatton, T.M. Dexter, M.G. Castro, and P.R. Lowenstein Uptake of alpha-(l)-iduronidase produced by retrovirally transduced fibroblasts into neuronal and glial cells in vitro Gene Ther. 4 1997 63 75
-
(1997)
Gene Ther.
, vol.4
, pp. 63-75
-
-
Stewart, K.1
Brown, O.A.2
Morelli, A.E.3
Fairbairn, L.J.4
Lashford, L.S.5
Cooper, A.6
Hatton, C.E.7
Dexter, T.M.8
Castro, M.G.9
Lowenstein, P.R.10
-
32
-
-
0028449393
-
Overexpression of the human lysosomal enzyme alpha-l-iduronidase in Chinese hamster ovary cells
-
E.D. Kakkis, A. Matynia, A.J. Jonas, and E.F. Neufeld Overexpression of the human lysosomal enzyme alpha-l-iduronidase in Chinese hamster ovary cells Protein Expr. Purif. 5 1994 225 232
-
(1994)
Protein Expr. Purif.
, vol.5
, pp. 225-232
-
-
Kakkis, E.D.1
Matynia, A.2
Jonas, A.J.3
Neufeld, E.F.4
-
33
-
-
4944239910
-
Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I
-
E. Kakkis, M. McEntee, C. Vogler, S. Le, B. Levy, P. Belichenko, W. Mobley, P. Dickson, S. Hanson, and M. Passage Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I Mol. Genet. Metab. 83 2004 163 174
-
(2004)
Mol. Genet. Metab.
, vol.83
, pp. 163-174
-
-
Kakkis, E.1
McEntee, M.2
Vogler, C.3
Le, S.4
Levy, B.5
Belichenko, P.6
Mobley, W.7
Dickson, P.8
Hanson, S.9
Passage, M.10
-
34
-
-
0030221033
-
Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I
-
E.D. Kakkis, M.F. McEntee, A. Schmidtchen, E.F. Neufeld, D.A. Ward, R.E. Gompf, S. Kania, C. Bedolla, S.L. Chien, and R.M. Shull Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I Biochem. Mol. Med. 58 1996 156 167
-
(1996)
Biochem. Mol. Med.
, vol.58
, pp. 156-167
-
-
Kakkis, E.D.1
McEntee, M.F.2
Schmidtchen, A.3
Neufeld, E.F.4
Ward, D.A.5
Gompf, R.E.6
Kania, S.7
Bedolla, C.8
Chien, S.L.9
Shull, R.M.10
-
35
-
-
0031149479
-
Hyperplastic changes within the leptomeninges of the rat and monkey in response to chronic intracerebroventricular infusion of nerve growth factor
-
P.A. Day-Lollini, G.R. Stewart, M.J. Taylor, R.M. Johnson, and G.J. Chellman Hyperplastic changes within the leptomeninges of the rat and monkey in response to chronic intracerebroventricular infusion of nerve growth factor Exp. Neurol. 145 1997 24 37
-
(1997)
Exp. Neurol.
, vol.145
, pp. 24-37
-
-
Day-Lollini, P.A.1
Stewart, G.R.2
Taylor, M.J.3
Johnson, R.M.4
Chellman, G.J.5
-
36
-
-
0025868787
-
Basic fibroblast growth factor prevents thalamic degeneration after cortical infarction
-
K. Yamada, A. Kinoshita, E. Kohmura, T. Sakaguchi, J. Taguchi, K. Kataoka, and T. Hayakawa Basic fibroblast growth factor prevents thalamic degeneration after cortical infarction J. Cereb. Blood Flow Metab. 11 1991 472 478
-
(1991)
J. Cereb. Blood Flow Metab.
, vol.11
, pp. 472-478
-
-
Yamada, K.1
Kinoshita, A.2
Kohmura, E.3
Sakaguchi, T.4
Taguchi, J.5
Kataoka, K.6
Hayakawa, T.7
-
37
-
-
0037435511
-
Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD
-
J.G. Nutt, K.J. Burchiel, C.L. Comella, J. Jankovic, A.E. Lang, E.R. Laws, A.M. Lozano, R.D. Penn, R.K. Simpson, M. Stacy, G.F. Wooten, and the ICV GDNF Study Group, Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD, Neurol. 60 (2003) 69-73.
-
(2003)
Neurol.
, vol.60
, pp. 69-73
-
-
Nutt, J.G.1
Burchiel, K.J.2
Comella, C.L.3
Jankovic, J.4
Lang, A.E.5
Laws, E.R.6
Lozano, A.M.7
Penn, R.D.8
Simpson, R.K.9
Stacy, M.10
Wooten, G.F.11
|