-
1
-
-
0028090049
-
Transfusion therapy in congenital coagulopathies
-
Lusher J, Transfusion therapy in congenital coagulopathies. Hematol Oncol Clin N Am 1994; 8: 1167-1180.
-
(1994)
Hematol Oncol Clin N Am
, vol.8
, pp. 1167-1180
-
-
Lusher, J.1
-
2
-
-
10144234803
-
Recombinant human factor IX: Replacement therapy, prophylaxis, and pharmacokinetics in canine hemophilia B
-
Brinkhous K et al. Recombinant human factor IX: replacement therapy, prophylaxis, and pharmacokinetics in canine hemophilia B. Blood 1996; 88: 2603-2610.
-
(1996)
Blood
, vol.88
, pp. 2603-2610
-
-
Brinkhous, K.1
-
3
-
-
0031846936
-
Hepatitis A virus infections associated with clotting factor concentrate in the United States
-
Soucie J et al. Hepatitis A virus infections associated with clotting factor concentrate in the United States. Transfusion 1998; 38: 573-579.
-
(1998)
Transfusion
, vol.38
, pp. 573-579
-
-
Soucie, J.1
-
4
-
-
0345718307
-
The treatment of hemophilia and related bleeding disorders, Medical Advisory No. 312
-
New York
-
National Hemophilia Foundation. The treatment of hemophilia and related bleeding disorders, Medical Advisory No. 312. In: Medical and Scientific Advisory Council (MASAC) Recommendations. New York, 1998, p 11-34.
-
(1998)
Medical and Scientific Advisory Council (MASAC) Recommendations
, pp. 11-34
-
-
-
5
-
-
0030910204
-
Haemophilia prophylaxis in young patients - A long-term follow-up
-
Lofqvist T, Nilsson I, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients - a long-term follow-up. J Intern Med 1997; 241: 395-400.
-
(1997)
J Intern Med
, vol.241
, pp. 395-400
-
-
Lofqvist, T.1
Nilsson, I.2
Berntorp, E.3
Pettersson, H.4
-
7
-
-
0027686238
-
In vivo gene therapy of hemophilia B: Sustained partial correction in factor IX-deficient
-
Kay M et al. In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient. Science 1993; 262: 117-119.
-
(1993)
Science
, vol.262
, pp. 117-119
-
-
Kay, M.1
-
8
-
-
0028934815
-
Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression
-
Dai Y et al. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression. Prof Nat Acad Sci USA 1995; 92: 1401-1405.
-
(1995)
Prof Nat Acad Sci USA
, vol.92
, pp. 1401-1405
-
-
Dai, Y.1
-
9
-
-
0032190675
-
Adeno-associated virus expression system for gene transfer
-
Rabinowitz J, Samulski J. Adeno-associated virus expression system for gene transfer. Curr Opin Biotechnol 1998; 9: 470-475.
-
(1998)
Curr Opin Biotechnol
, vol.9
, pp. 470-475
-
-
Rabinowitz, J.1
Samulski, J.2
-
10
-
-
0023081188
-
Adeno-associated viruses: An update
-
Berns K, Bohenzky R. Adeno-associated viruses: an update. Adv Virus Res 1987; 32: 243-306.
-
(1987)
Adv Virus Res
, vol.32
, pp. 243-306
-
-
Berns, K.1
Bohenzky, R.2
-
11
-
-
9244234931
-
In vivo model of adeno-associated virus vector persistence and rescue
-
Afione S et al. In vivo model of adeno-associated virus vector persistence and rescue. J Virol 1996; 70: 3235-3241.
-
(1996)
J Virol
, vol.70
, pp. 3235-3241
-
-
Afione, S.1
-
12
-
-
0027484584
-
Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector
-
Flotte T et al. Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci USA 1993; 90: 10613-10617.
-
(1993)
Proc Natl Acad Sci USA
, vol.90
, pp. 10613-10617
-
-
Flotte, T.1
-
13
-
-
0029843596
-
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
-
Xiao X, Li J, Samulski R. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996; 70: 8098-8108.
-
(1996)
J Virol
, vol.70
, pp. 8098-8108
-
-
Xiao, X.1
Li, J.2
Samulski, R.3
-
14
-
-
0029151404
-
Adeno-associated virus vectors for gene therapy
-
Flotte T, Carter B. Adeno-associated virus vectors for gene therapy. Gene Therapy 1995; 2: 357-362.
-
(1995)
Gene Therapy
, vol.2
, pp. 357-362
-
-
Flotte, T.1
Carter, B.2
-
15
-
-
0029849257
-
Quantitative analysis of the packaging capacity of recombinant adeno-associated virus
-
Dong J, Fan P, Frizzell R. Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum Gene Ther 1996; 7: 2101-2112.
-
(1996)
Hum Gene Ther
, vol.7
, pp. 2101-2112
-
-
Dong, J.1
Fan, P.2
Frizzell, R.3
-
16
-
-
0030970938
-
Recombinant adeno-associated viral vectors mediate long-term transgene expression in muscle
-
Clark K, Sferra T, Johnson P. Recombinant adeno-associated viral vectors mediate long-term transgene expression in muscle. Hum Gene Ther 1997; 8: 659-669.
-
(1997)
Hum Gene Ther
, vol.8
, pp. 659-669
-
-
Clark, K.1
Sferra, T.2
Johnson, P.3
-
17
-
-
0026511128
-
Expression of human factor IX in mice after injection of genetically modified myoblasts
-
Yao S, Kurachi K. Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc Natl Acad Sci USA 1992; 89: 3357-3361.
-
(1992)
Proc Natl Acad Sci USA
, vol.89
, pp. 3357-3361
-
-
Yao, S.1
Kurachi, K.2
-
18
-
-
0030902985
-
Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
-
Herzog R et al. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc Nat Acad Sci USA 1997; 94: 5804-5809.
-
(1997)
Proc Nat Acad Sci USA
, vol.94
, pp. 5804-5809
-
-
Herzog, R.1
-
19
-
-
0024805130
-
Canine hemophilia B resulting from a point mutation with unusual consequences
-
Evans J et al. Canine hemophilia B resulting from a point mutation with unusual consequences. Proc Nat Acad Sci USA 1989; 86: 10095-10099.
-
(1989)
Proc Nat Acad Sci USA
, vol.86
, pp. 10095-10099
-
-
Evans, J.1
-
20
-
-
0030962380
-
Extravascular administration of factor IX: Potential for replacement therapy of canine and human hemophilia B
-
Liles D et al. Extravascular administration of factor IX: potential for replacement therapy of canine and human hemophilia B. Thromb Haemostas 1997; 77: 944-948.
-
(1997)
Thromb Haemostas
, vol.77
, pp. 944-948
-
-
Liles, D.1
-
21
-
-
0031983639
-
Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia
-
Monahan P et al. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Therapy 1998; 5: 40-49.
-
(1998)
Gene Therapy
, vol.5
, pp. 40-49
-
-
Monahan, P.1
-
22
-
-
0028900484
-
Role of intron I in expression of the human factor IX gene
-
Kurachi S, Hitomi Y, Furukawa M, Kurachi K. Role of intron I in expression of the human factor IX gene. J Biol Chem 1995; 270: 5276-5281.
-
(1995)
J Biol Chem
, vol.270
, pp. 5276-5281
-
-
Kurachi, S.1
Hitomi, Y.2
Furukawa, M.3
Kurachi, K.4
-
23
-
-
2642642141
-
Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus
-
Xiao X, Li J, Samulski R. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 1998; 72: 2224-2232.
-
(1998)
J Virol
, vol.72
, pp. 2224-2232
-
-
Xiao, X.1
Li, J.2
Samulski, R.3
-
24
-
-
0024313169
-
Molecular cloning of a cDNA encoding canine factor IX
-
Evans J et al. Molecular cloning of a cDNA encoding canine factor IX. Blood 1989; 74: 207-212.
-
(1989)
Blood
, vol.74
, pp. 207-212
-
-
Evans, J.1
-
25
-
-
0025321444
-
Human parvoviruses
-
Anderson L, Human parvoviruses. J Infect Dis 1990; 161: 603-608.
-
(1990)
J Infect Dis
, vol.161
, pp. 603-608
-
-
Anderson, L.1
-
26
-
-
0029923638
-
Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors
-
Ferrari F, Samulski T, Shenk T, Samulski R. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol 1996; 70: 3227-3234.
-
(1996)
J Virol
, vol.70
, pp. 3227-3234
-
-
Ferrari, F.1
Samulski, T.2
Shenk, T.3
Samulski, R.4
-
27
-
-
0031011667
-
Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus
-
Flannery J et al. Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proc Natl Acad Sci USA 1997; 94: 6916-6921.
-
(1997)
Proc Natl Acad Sci USA
, vol.94
, pp. 6916-6921
-
-
Flannery, J.1
-
28
-
-
0026635406
-
Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B
-
Nilsson I, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992; 232: 25.
-
(1992)
J Intern Med
, vol.232
, pp. 25
-
-
Nilsson, I.1
Berntorp, E.2
Lofqvist, T.3
Pettersson, H.4
-
29
-
-
17444387438
-
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
-
Herzog R et al. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nature Med 1999; 5: 56-63.
-
(1999)
Nature Med
, vol.5
, pp. 56-63
-
-
Herzog, R.1
-
30
-
-
17444372024
-
Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
-
Snyder R et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nature Med 1999; 5: 64-70.
-
(1999)
Nature Med
, vol.5
, pp. 64-70
-
-
Snyder, R.1
-
31
-
-
0030997346
-
Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
-
Snyder R et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet 1997; 16: 270-276.
-
(1997)
Nat Genet
, vol.16
, pp. 270-276
-
-
Snyder, R.1
-
32
-
-
0031906147
-
Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions
-
Summerford C, Samulski R. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 1998; 72: 1438-1445.
-
(1998)
J Virol
, vol.72
, pp. 1438-1445
-
-
Summerford, C.1
Samulski, R.2
-
33
-
-
0032589751
-
AlphaVbeta5 integrin: A co-receptor for adeno-associated virus type 2 infection
-
Summerford C, Bartlett J, Samulski R. AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection. Nature Med 1999; 5: 78-82.
-
(1999)
Nature Med
, vol.5
, pp. 78-82
-
-
Summerford, C.1
Bartlett, J.2
Samulski, R.3
-
34
-
-
0033010884
-
Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2
-
Qing K et al. Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nature Med 1999; 5: 71-77.
-
(1999)
Nature Med
, vol.5
, pp. 71-77
-
-
Qing, K.1
-
35
-
-
0032917854
-
Viral receptors and vector purification: New approaches for generating clinical-grade reagents
-
Summerford C, Samulski R. Viral receptors and vector purification: new approaches for generating clinical-grade reagents. Nature Med 1999; 5: 587.
-
(1999)
Nature Med
, vol.5
, pp. 587
-
-
Summerford, C.1
Samulski, R.2
-
36
-
-
0030841312
-
Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles
-
Chiorini J et al. Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles. J Virol 1997; 71: 6823-6833.
-
(1997)
J Virol
, vol.71
, pp. 6823-6833
-
-
Chiorini, J.1
-
37
-
-
0030451214
-
Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein
-
Kessler P et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 1996; 93: 14082-14087.
-
(1996)
Proc Natl Acad Sci USA
, vol.93
, pp. 14082-14087
-
-
Kessler, P.1
-
38
-
-
0031058024
-
Recombinant adeno-associated virus for muscle directed gene therapy
-
Fisher K et al. Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med 1997; 3: 306-312.
-
(1997)
Nature Med
, vol.3
, pp. 306-312
-
-
Fisher, K.1
-
39
-
-
0029655894
-
Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis
-
Fisher K et al. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol 1996; 70: 520-532.
-
(1996)
J Virol
, vol.70
, pp. 520-532
-
-
Fisher, K.1
-
40
-
-
0031691257
-
Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term persistence in muscle tissue
-
Duan D et al. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term persistence in muscle tissue. J Virol 1998; 72: 8568-8577.
-
(1998)
J Virol
, vol.72
, pp. 8568-8577
-
-
Duan, D.1
-
41
-
-
0031901455
-
Cellular proteins required for adeno-associated virus DNA replication in the absence of adenovirus coinfection
-
Ni T et al. Cellular proteins required for adeno-associated virus DNA replication in the absence of adenovirus coinfection. J Virol 1998; 72: 2777-2787.
-
(1998)
J Virol
, vol.72
, pp. 2777-2787
-
-
Ni, T.1
-
42
-
-
0033023881
-
Structure of adeno-associated virus vector DNA following transduction of the skeletal muscle
-
Vincent-Lacaze N et al. Structure of adeno-associated virus vector DNA following transduction of the skeletal muscle. J Virol 1999; 73: 1949-1955.
-
(1999)
J Virol
, vol.73
, pp. 1949-1955
-
-
Vincent-Lacaze, N.1
-
43
-
-
0030857125
-
Human factor IX binds to specific sites on the collagenous domain of collagen IV
-
Wolberg A, Stafford D, Erie D. Human factor IX binds to specific sites on the collagenous domain of collagen IV. J Biol Chem 1997; 272: 16717-16720.
-
(1997)
J Biol Chem
, vol.272
, pp. 16717-16720
-
-
Wolberg, A.1
Stafford, D.2
Erie, D.3
-
45
-
-
0032907636
-
Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector
-
Greelish J et al. Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector. Nature Med 1999; 5: 439.
-
(1999)
Nature Med
, vol.5
, pp. 439
-
-
Greelish, J.1
-
46
-
-
0031278324
-
New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors
-
Ferrari F, Xiao X, McCarty D, Samulski R. New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors. Nature Med 1997; 3: 1295-1297.
-
(1997)
Nature Med
, vol.3
, pp. 1295-1297
-
-
Ferrari, F.1
Xiao, X.2
McCarty, D.3
Samulski, R.4
-
47
-
-
0014202537
-
Selective extraction of polyoma DNA from infected mouse cell cultures
-
Hirt B. Selective extraction of polyoma DNA from infected mouse cell cultures. J Mol Biol 1967; 26: 365-369.
-
(1967)
J Mol Biol
, vol.26
, pp. 365-369
-
-
Hirt, B.1
-
49
-
-
0000386558
-
Analysis of RNA by Northern and slot blot hybridization
-
Ausubel F et al (eds). John Wiley & Sons
-
Kingston R. Analysis of RNA by Northern and slot blot hybridization. In: Ausubel F et al (eds). Current Protocols in Molecular Biology. John Wiley & Sons: 1997, pp 4.9.1-4.9.13.
-
(1997)
Current Protocols in Molecular Biology
, pp. 491-4913
-
-
Kingston, R.1
|