-
1
-
-
79955405275
-
Good manufacturing practice production of self-complementary serotype 8 adeno-Associated viral vector for a hemophilia B clinical trial
-
Allay, J.A., Sleep, S., Long, S., et al. (2011). Good manufacturing practice production of self-complementary serotype 8 adeno-Associated viral vector for a hemophilia B clinical trial. Hum. Gene Ther. 22, 595-604
-
(2011)
Hum. Gene Ther
, vol.22
, pp. 595-604
-
-
Allay, J.A.1
Sleep, S.2
Long, S.3
-
2
-
-
44249120315
-
Effect of gene therapy on visual function in Leber's congenital amaurosis
-
Bainbridge, J.W., Smith, A.J., Barker, S.S., et al. (2008). Effect of gene therapy on visual function in Leber's congenital amaurosis. N. Engl. J. Med. 358, 2231-2239
-
(2008)
N. Engl. J. Med
, vol.358
, pp. 2231-2239
-
-
Bainbridge, J.W.1
Smith, A.J.2
Barker, S.S.3
-
3
-
-
39849110435
-
Reversal of blindness in animal models of Leber congenital amaurosis using optimized AAV2-mediated gene transfer
-
Bennicelli, J., Wright, J.F., Komaromy, A., et al. (2008). Reversal of blindness in animal models of Leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol. Ther. 16, 458-465
-
(2008)
Mol. Ther
, vol.16
, pp. 458-465
-
-
Bennicelli, J.1
Wright, J.F.2
Komaromy, A.3
-
4
-
-
73449128979
-
Safety and tolerability of putaminal aadc gene therapy for parkinson disease
-
Christine, C.W., Starr, P.A., Larson, P.S., et al. (2009). Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology 73, 1662-1669
-
(2009)
Neurology
, vol.73
, pp. 1662-1669
-
-
Christine, C.W.1
Starr, P.A.2
Larson, P.S.3
-
5
-
-
0033541640
-
Highly purified recombinant adeno-Associated virus vectors are biologically active and free of detectable helper and wildtype viruses
-
Clark, K.R., Liu, X., McGrath, J.P., and Johnson, P.R. (1999). Highly purified recombinant adeno-Associated virus vectors are biologically active and free of detectable helper and wildtype viruses. Hum. Gene Ther. 10, 1031-1039
-
(1999)
Hum. Gene Ther
, vol.10
, pp. 1031-1039
-
-
Clark, K.R.1
Liu, X.2
McGrath, J.P.3
Johnson, P.R.4
-
6
-
-
84873498637
-
Gene therapies advance, but some see manufacturing challenges
-
Dolgin, E. (2012). Gene therapies advance, but some see manufacturing challenges. Nat. Med. 18, 1718-1719
-
(2012)
Nat. Med
, vol.18
, pp. 1718-1719
-
-
Dolgin, E.1
-
7
-
-
44949222522
-
Results from a phase I safety trial of hAADC gene therapy for Parkinson disease
-
Eberling, J.L., Jagust, W.J., Christine, C.W., et al. (2008). Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology 70, 1980-1983
-
(2008)
Neurology
, vol.70
, pp. 1980-1983
-
-
Eberling, J.L.1
Jagust, W.J.2
Christine, C.W.3
-
8
-
-
84865245416
-
Systemic errors in quantitative polymerase chain reaction titration of selfcomplementary adeno-Associated viral vectors and improved alternative methods
-
Fagone, P., Wright, J.F., Nathwani, A.C., et al. (2011). Systemic errors in quantitative polymerase chain reaction titration of selfcomplementary adeno-Associated viral vectors and improved alternative methods. Hum. Gene Ther. Methods 23, 1-7
-
(2011)
Hum. Gene Ther. Methods
, vol.23
, pp. 1-7
-
-
Fagone, P.1
Wright, J.F.2
Nathwani, A.C.3
-
9
-
-
54949104686
-
Treatment of leber congenital amaurosis due to rpe65 mutations by ocular subretinal injection of adeno-Associated virus gene vector: Short-Term results of a phase i trial
-
Hauswirth, W.W., Aleman, T.S., Kaushal, S., et al. (2008). Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-Associated virus gene vector: Short-Term results of a phase I trial. Hum. Gene Ther. 19, 979-990
-
(2008)
Hum. Gene Ther
, vol.19
, pp. 979-990
-
-
Hauswirth, W.W.1
Aleman, T.S.2
Kaushal, S.3
-
10
-
-
81255175501
-
Highthroughput droplet digital pcr system for absolute quantitation of dna copy number
-
Hindson, B.J., Ness, K.D., Masquelier, D.A., et al. (2011). Highthroughput droplet digital PCR system for absolute quantitation of DNA copy number. Anal. Chem. 83, 8604-8610
-
(2011)
Anal. Chem
, vol.83
, pp. 8604-8610
-
-
Hindson, B.J.1
Ness, K.D.2
Masquelier, D.A.3
-
11
-
-
84865402788
-
Molecular quantification of environmental DNA using microfluidics and digital PCR
-
Hoshino, T., and Inagaki, F. (2012). Molecular quantification of environmental DNA using microfluidics and digital PCR. Syst. Appl. Microbiol. 35, 390-395
-
(2012)
Syst. Appl. Microbiol
, vol.35
, pp. 390-395
-
-
Hoshino, T.1
Inagaki, F.2
-
12
-
-
34250683023
-
Safety and tolerability of gene therapy with an adeno-Associated virus (aav) borne gad gene for parkinson's disease: An open label, phase i trial
-
Kaplitt, M.G., Feigin, A., Tang, C., et al. (2007). Safety and tolerability of gene therapy with an adeno-Associated virus (AAV) borne GAD gene for Parkinson's disease: An open label, phase I trial. Lancet 369, 2097-2105
-
(2007)
Lancet
, vol.369
, pp. 2097-2105
-
-
Kaplitt, M.G.1
Feigin, A.2
Tang, C.3
-
13
-
-
84862969739
-
Native molecular state of adeno-Associated viral vectors revealed by single-molecule sequencing
-
Kapranov, P., Chen, L., Dederich, D., et al. (2012). Native molecular state of adeno-Associated viral vectors revealed by single-molecule sequencing. Hum. Gene Ther. 23, 46-55
-
(2012)
Hum. Gene Ther
, vol.23
, pp. 46-55
-
-
Kapranov, P.1
Chen, L.2
Dederich, D.3
-
14
-
-
77958104835
-
Rapid, simple, and versatile manufacturing of recombinant adeno-Associated viral vectors at scale
-
Lock, M., Alvira, M., Vandenberghe, L.H., et al. (2010a). Rapid, simple, and versatile manufacturing of recombinant adeno-Associated viral vectors at scale. Hum. Gene Ther. 21, 1259-1271
-
(2010)
Hum. Gene Ther
, vol.21
, pp. 1259-1271
-
-
Lock, M.1
Alvira, M.2
Vandenberghe, L.H.3
-
15
-
-
77958099436
-
Characterization of a recombinant adeno-Associated virus type 2 reference standardmaterial
-
Lock, M., McGorray, S., Auricchio, A., et al. (2010b). Characterization of a recombinant adeno-Associated virus type 2 Reference StandardMaterial. Hum. Gene Ther. 21, 1273-1285
-
(2010)
Hum. Gene Ther
, vol.21
, pp. 1273-1285
-
-
Lock, M.1
McGorray, S.2
Auricchio, A.3
-
16
-
-
84872030621
-
Analysis of particle content of recombinant adeno-Associated virus serotype 8 vectors by ion-exchange chromatography
-
Lock, M., Alvira, M.R., and Wilson, J.M. (2012). Analysis of particle content of recombinant adeno-Associated virus serotype 8 vectors by ion-exchange chromatography. Hum. Gene Ther. Methods 23, 56-64
-
(2012)
Hum. Gene Ther. Methods
, vol.23
, pp. 56-64
-
-
Lock, M.1
Alvira, M.R.2
Wilson, J.M.3
-
17
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
Maguire, A.M., Simonelli, F., Pierce, E.A., et al. (2008). Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med. 358, 2240-2248
-
(2008)
N. Engl. J. Med
, vol.358
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
-
18
-
-
70350620424
-
Age-dependent effects of rpe65 gene therapy for leber's congenital amaurosis: A phase 1 dose-escalation trial
-
Maguire AM, High KA, Auricchio A, Wright JF, et al. (2009). Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: A phase 1 dose-escalation trial. Lancet 374, 1597-605
-
(2009)
Lancet
, vol.374
, pp. 1597-1605
-
-
Maguire, A.M.1
High, K.A.2
Auricchio, A.3
Wright, J.F.4
-
19
-
-
84855982119
-
Evaluation of a droplet digital polymerase chain reaction format for DNA copy number quantification
-
Pinheiro, L.B., Coleman, V.A., Hindson, C.M., et al. (2012). Evaluation of a droplet digital polymerase chain reaction format for DNA copy number quantification. Anal. Chem. 84, 1003-1011
-
(2012)
Anal. Chem
, vol.84
, pp. 1003-1011
-
-
Pinheiro, L.B.1
Coleman, V.A.2
Hindson, C.M.3
-
20
-
-
0024311525
-
Helper-free stocks of recombinant adeno-Associated viruses: Normal integration does not require viral gene expression
-
Samulski, R.J., Chang, L.S., and Shenk, T. (1989). Helper-free stocks of recombinant adeno-Associated viruses: Normal integration does not require viral gene expression. J. Virol. 63, 3822-3828
-
(1989)
J. Virol
, vol.63
, pp. 3822-3828
-
-
Samulski, R.J.1
Chang, L.S.2
Shenk, T.3
-
21
-
-
80052314136
-
Evaluation of digital PCR for absolute DNA quantification
-
Sanders, R., Huggett, J.F., Bushell, C.A., et al. (2011). Evaluation of digital PCR for absolute DNA quantification. Anal. Chem. 83, 6474-6484
-
(2011)
Anal. Chem
, vol.83
, pp. 6474-6484
-
-
Sanders, R.1
Huggett, J.F.2
Bushell, C.A.3
-
22
-
-
84870758826
-
Viral diagnostics in the era of digital polymerase chain reaction
-
Sedlak, R.H., and Jerome, K.R. (2013). Viral diagnostics in the era of digital polymerase chain reaction. Diagn. Microbiol. Infect. Dis. 75, 1-4
-
(2013)
Diagn. Microbiol. Infect. Dis
, vol.75
, pp. 1-4
-
-
Sedlak, R.H.1
Jerome, K.R.2
-
23
-
-
0026537986
-
Identification of ras oncogene mutations in the stool of patients with curable colorectal tumors
-
Sidransky, D., Tokino, T., Hamilton, S.R., et al. (1992). Identification of ras oncogene mutations in the stool of patients with curable colorectal tumors. Science 256, 102-105
-
(1992)
Science
, vol.256
, pp. 102-105
-
-
Sidransky, D.1
Tokino, T.2
Hamilton, S.R.3
-
24
-
-
77649242176
-
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration
-
Simonelli, F., Maguire, A.M., Testa, F., et al. (2010). Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol. Ther. 18, 643-650
-
(2010)
Mol. Ther
, vol.18
, pp. 643-650
-
-
Simonelli, F.1
Maguire, A.M.2
Testa, F.3
-
25
-
-
0037223085
-
Quantification of adeno-Associated virus particles and empty capsids by optical density measurement
-
Sommer, J.M., Smith, P.H., Parthasarathy, S., et al. (2003). Quantification of adeno-Associated virus particles and empty capsids by optical density measurement. Mol. Ther. 7, 122-128
-
(2003)
Mol. Ther
, vol.7
, pp. 122-128
-
-
Sommer, J.M.1
Smith, P.H.2
Parthasarathy, S.3
-
27
-
-
84872233239
-
Limitations of encapsidation of recombinant self-complementary adenoassociated viral genomes in different serotype capsids and their quantitation
-
Wang, Y., Ling, C., Song, L., et al. (2012). Limitations of encapsidation of recombinant self-complementary adenoassociated viral genomes in different serotype capsids and their quantitation. Hum. Gene Ther. Methods 23, 225-233
-
(2012)
Hum. Gene Ther. Methods
, vol.23
, pp. 225-233
-
-
Wang, Y.1
Ling, C.2
Song, L.3
|