-
1
-
-
70349229299
-
Coagulation disorders
-
Loscalzo J, Kasper D, HauserS, Fauci A, Jameson J, Longo D, eds, 19th Edition: McGraw Hill Education
-
Arruda V, High KA. Coagulation Disorders. In: Loscalzo J, Kasper D, HauserS, Fauci A, Jameson J, Longo D, eds. Harrison's Principles of Internal Medicine 19th Edition: McGraw Hill Education; 2015.
-
(2015)
Harrison's Principles of Internal Medicine
-
-
Arruda, V.1
High, K.A.2
-
2
-
-
0033028402
-
Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized
-
10554828
-
Astermark J, Petrini P, Tengborn L, Schulman S, Ljung R, Berntorp E. Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized. Br J Haematol. 1999;105(4):1109-1113. PMID: 10554828
-
(1999)
Br J Haematol.
, vol.105
, Issue.4
, pp. 1109-1113
-
-
Astermark, J.1
Petrini, P.2
Tengborn, L.3
Schulman, S.4
Ljung, R.5
Berntorp, E.6
-
3
-
-
34547757915
-
Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia
-
17687129
-
Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N EnglJ Med. 2007;357(6):535-544. PMID: 17687129
-
(2007)
N EnglJ Med
, vol.357
, Issue.6
, pp. 535-544
-
-
Manco-Johnson, M.J.1
Abshire, T.C.2
Shapiro, A.D.3
Riske, B.4
Hacker, M.R.5
Kilcoyne, R.6
-
4
-
-
84926139661
-
Optimal treatment strategies for hemophilia: Achievements and limitations of current prophylactic regimens
-
25712992
-
Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood. 2015;125(13):2038-2044. doi: 10.1182/blood-2015-01-528414 PMID: 25712992
-
(2015)
Blood
, vol.125
, Issue.13
, pp. 2038-2044
-
-
Oldenburg, J.1
-
5
-
-
0036147943
-
The North American immune tolerance registry: Practices, outcomes, outcome predictors
-
11848456
-
Di Michele DM, Kroner BL. The North American Immune Tolerance Registry: practices, outcomes, outcome predictors. Thromb Haemost. 2002;87(1):52-57. PMID: 11848456
-
(2002)
Thromb Haemost
, vol.87
, Issue.1
, pp. 52-57
-
-
Di Michele, D.M.1
Kroner, B.L.2
-
6
-
-
84856866914
-
The principal results of the International Immune Tolerance Study: A randomized dose comparison
-
22101900
-
Hay CR, Di Michele DM. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood. 2012;119(6):1335-1344. doi: 10.1182/blood-2011-08-369132 PMID: 22101900
-
(2012)
Blood
, vol.119
, Issue.6
, pp. 1335-1344
-
-
Hay, C.R.1
Di Michele, D.M.2
-
7
-
-
34249690732
-
Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: The CANAL cohort study
-
17289808
-
Gouw SC, van der Bom JG, Marijkevan den Berg H. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood. 2007;109(11):4648-4654. PMID: 17289808
-
(2007)
Blood
, vol.109
, Issue.11
, pp. 4648-4654
-
-
Gouw, S.C.1
Van Der Bom, J.G.2
Marijkevan Den Berg, H.3
-
8
-
-
0038383605
-
The epidemiology of inhibitors in haemophilia A: A systematic review
-
12828678
-
Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia. 2003;9(4):418-435. PMID: 12828678
-
(2003)
Haemophilia
, vol.9
, Issue.4
, pp. 418-435
-
-
Wight, J.1
Paisley, S.2
-
9
-
-
0034070492
-
Establishing haemophilia care in developing countries: Using data to overcome the barrier of pessimism
-
10792469
-
Evatt BL, Robillard L. Establishing haemophilia care in developing countries: using data to overcome the barrier of pessimism. Haemophilia. 2000;6(3):131-134. PMID: 10792469
-
(2000)
Haemophilia
, vol.6
, Issue.3
, pp. 131-134
-
-
Evatt, B.L.1
Robillard, L.2
-
10
-
-
33644820684
-
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
-
16474400
-
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJE, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med. 2006;12(3):342-347. PMID: 16474400
-
(2006)
Nat Med
, vol.12
, Issue.3
, pp. 342-347
-
-
Manno, C.S.1
Pierce, G.F.2
Arruda, V.R.3
Glader, B.4
Ragni, M.5
Rasko, J.J.E.6
-
11
-
-
0037089331
-
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
-
11929752
-
Mount JD, Herzog RW, Tillson DM, Goodman SA, Robinson N, McCleland ML, et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood. 2002;99(8):2670-2676. PMID: 11929752
-
(2002)
Blood
, vol.99
, Issue.8
, pp. 2670-2676
-
-
Mount, J.D.1
Herzog, R.W.2
Tillson, D.M.3
Goodman, S.A.4
Robinson, N.5
McCleland, M.L.6
-
12
-
-
84911383748
-
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
-
25409372
-
Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N EnglJ Med. 2014;371(21):1994-2004. doi: 10.1056/NEJMoa1407309 PMID: 25409372
-
(2014)
N EnglJ Med
, vol.371
, Issue.21
, pp. 1994-2004
-
-
Nathwani, A.C.1
Reiss, U.M.2
Tuddenham, E.G.3
Rosales, C.4
Chowdary, P.5
McIntosh, J.6
-
13
-
-
66149110999
-
Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency
-
19293459
-
Nichols TC, Dillow AM, Franck HW, Merricks EP, Raymer RA, Bellinger DA, et al. Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency. ILAR Journal. 2009;50(2):144-167. PMID: 19293459
-
(2009)
ILAR Journal.
, vol.50
, Issue.2
, pp. 144-167
-
-
Nichols, T.C.1
Dillow, A.M.2
Franck, H.W.3
Merricks, E.P.4
Raymer, R.A.5
Bellinger, D.A.6
-
14
-
-
78650647673
-
Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy
-
20876851
-
Finn JD, Ozelo MC, Sabatino DE, Franck HWG, Merricks EP, Crudele JM, et al. Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy. Blood. 2010;116(26):5842-5848. doi: 10.1182/blood-2010-06-288001 PMID: 20876851
-
(2010)
Blood
, vol.116
, Issue.26
, pp. 5842-5848
-
-
Finn, J.D.1
Ozelo, M.C.2
Sabatino, D.E.3
Franck, H.W.G.4
Merricks, E.P.5
Crudele, J.M.6
-
15
-
-
84877704167
-
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
-
23426947
-
McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, et al. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood. 2013;121(17):3335-3344. doi: 10.1182/blood-2012-10-462200 PMID: 23426947
-
(2013)
Blood
, vol.121
, Issue.17
, pp. 3335-3344
-
-
McIntosh, J.1
Lenting, P.J.2
Rosales, C.3
Lee, D.4
Rabbanian, S.5
Raj, D.6
-
16
-
-
79952194475
-
Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors
-
21081906
-
Sabatino DE, Lange AM, Altynova ES, Sarkar R, Zhou S, Merricks EP, et al. Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors. Mol Ther. 2011;19(3):442-449. doi: 10.1038/mt.2010.240 PMID: 21081906
-
(2011)
Mol Ther
, vol.19
, Issue.3
, pp. 442-449
-
-
Sabatino, D.E.1
Lange, A.M.2
Altynova, E.S.3
Sarkar, R.4
Zhou, S.5
Merricks, E.P.6
-
17
-
-
84880427173
-
Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype
-
23372167
-
Siner JI, Iacobelli NP, Sabatino DE, Ivanciu L, Zhou S, Poncz M, et al. Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype. Blood. 2013;121(21):4396-4403. doi: 10.1182/blood-2012-10-464164 PMID: 23372167
-
(2013)
Blood
, vol.121
, Issue.21
, pp. 4396-4403
-
-
Siner, J.I.1
Iacobelli, N.P.2
Sabatino, D.E.3
Ivanciu, L.4
Zhou, S.5
Poncz, M.6
-
18
-
-
84901414179
-
Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy
-
24637359
-
Nair N, Rincon MY, Evens H, Evens H, Sarcar S, Dastidar S, et al. Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy. Blood. 2014;123(20):3195-3199. doi: 10.1182/blood-2013-10-534032 PMID: 24637359
-
(2014)
Blood
, vol.123
, Issue.20
, pp. 3195-3199
-
-
Nair, N.1
Rincon, M.Y.2
Evens, H.3
Evens, H.4
Sarcar, S.5
Dastidar, S.6
-
19
-
-
0021318961
-
Development offactor VIII: C antibodies in dogs with hemophilia A (factor VIII: C deficiency)
-
6419797
-
Giles AR, Tinlin S, Hoogendoorn H, Greenwood P, Greenwood R. Development offactor VIII: C antibodies in dogs with hemophilia A (factor VIII: C deficiency). Blood. 1984;63(2):451-456. PMID: 6419797
-
(1984)
Blood
, vol.63
, Issue.2
, pp. 451-456
-
-
Giles, A.R.1
Tinlin, S.2
Hoogendoorn, H.3
Greenwood, P.4
Greenwood, R.5
-
20
-
-
73349120945
-
Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model
-
19770361
-
Sabatino DE, Freguia CF, Toso R, Andrey Santos A, Merricks EP, Kazazian HH Jr, et al. Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model. Blood. 2009;114(20):4562-4565. doi: 10.1182/blood-2009-05-220327 PMID: 19770361
-
(2009)
Blood
, vol.114
, Issue.20
, pp. 4562-4565
-
-
Sabatino, D.E.1
Freguia, C.F.2
Toso, R.3
Andrey Santos, A.4
Merricks, E.P.5
Kazazian, H.H.6
-
21
-
-
84880559842
-
Overcoming preexisting humoral immunity to AAV using capsid decoys
-
Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, et al. Overcoming preexisting humoral immunity to AAV using capsid decoys. SciTransl Med. 2013;5(194):194ra192.
-
(2013)
SciTransl Med
, vol.5
, Issue.194
, pp. 194ra192
-
-
Mingozzi, F.1
Anguela, X.M.2
Pavani, G.3
Chen, Y.4
Davidson, R.J.5
Hui, D.J.6
-
22
-
-
33751181882
-
Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy
-
16868252
-
Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy D, Vargas JA, et al. Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood. 2006;108(10):3321-3328. PMID: 16868252
-
(2006)
Blood
, vol.108
, Issue.10
, pp. 3321-3328
-
-
Jiang, H.1
Couto, L.B.2
Patarroyo-White, S.3
Liu, T.4
Nagy, D.5
Vargas, J.A.6
-
23
-
-
80052496589
-
Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors
-
21476868
-
Wang L, Calcedo R, Bell P, Lin J, Grant RL, Siegel DL, et al. Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors. Hum Gene Ther. 2011;22(11):1389-1401. doi: 10.1089/hum.2011.031 PMID: 21476868
-
(2011)
Hum Gene Ther.
, vol.22
, Issue.11
, pp. 1389-1401
-
-
Wang, L.1
Calcedo, R.2
Bell, P.3
Lin, J.4
Grant, R.L.5
Siegel, D.L.6
-
24
-
-
84924997217
-
AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
-
25568350
-
Crudele JM, Finn JD, Siner JI, Martin NB, Niemeyer GP, Zhou S, et al. AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice. Blood. 2015;125(10):1553-1561. doi: 10.1182/blood-2014-07-588194 PMID: 25568350
-
(2015)
Blood
, vol.125
, Issue.10
, pp. 1553-1561
-
-
Crudele, J.M.1
Finn, J.D.2
Siner, J.I.3
Martin, N.B.4
Niemeyer, G.P.5
Zhou, S.6
-
25
-
-
78149246105
-
WAG-F8 (m1Ycb) rats harboring a factor VIII gene mutation provide a new animal model for hemophilia A
-
20626616
-
Booth CJ, Brooks MB, Rockwell S, Murphy JW, Rinder HM, Zelterman D, et al. WAG-F8 (m1Ycb) rats harboring a factor VIII gene mutation provide a new animal model for hemophilia A. JThromb Haemost. 2010;8(11):2472-2477. doi: 10.1111/j.1538-7836.2010.03978.x PMID: 20626616
-
(2010)
JThromb Haemost
, vol.8
, Issue.11
, pp. 2472-2477
-
-
Booth, C.J.1
Brooks, M.B.2
Rockwell, S.3
Murphy, J.W.4
Rinder, H.M.5
Zelterman, D.6
-
26
-
-
74749088747
-
Clinical and molecular characterization of a re-established line of sheep exhibiting hemophilia A
-
19943872
-
Porada CD, Sanada C, Long CR, Wood A, Desai J, Frederick N, et al. Clinical and molecular characterization of a re-established line of sheep exhibiting hemophilia A. JThromb Haemost. 2010;8(2):276-285. doi: 10.1111/j.1538-7836.2009.03697.x PMID: 19943872
-
(2010)
JThromb Haemost
, vol.8
, Issue.2
, pp. 276-285
-
-
Porada, C.D.1
Sanada, C.2
Long, C.R.3
Wood, A.4
Desai, J.5
Frederick, N.6
-
27
-
-
84962121461
-
And Model Systems for Hemophilia A Gene Therapy
-
Mao J, Xi X, Kapranov P, Dong B, Firrman J, Xu R, et al. and Model Systems for Hemophilia A Gene Therapy. J Genet Syndr Gene Ther. 2013; Suppl 1:.
-
(2013)
J Genet Syndr Gene Ther.
-
-
Mao, J.1
Xi, X.2
Kapranov, P.3
Dong, B.4
Firrman, J.5
Xu, R.6
-
28
-
-
84899516238
-
Genetic testing in bleeding disorders
-
24762276
-
de Brasi C, El-Maarri O, Perry DJ, Oldenburg J, Pezeshkpoor B, Goodeve A. Genetic testing in bleeding disorders. Haemophilia. 2014;20 Suppl 4:54-58. doi: 10.1111/hae.12409 PMID: 24762276
-
(2014)
Haemophilia
, vol.20
, pp. 54-58
-
-
De Brasi, C.1
El-Maarri, O.2
Perry, D.J.3
Oldenburg, J.4
Pezeshkpoor, B.5
Goodeve, A.6
-
29
-
-
54149083581
-
Detection of large duplications within the factor VIII gene by MLPA
-
18752578
-
Rost S, Loffler S, Pavlova A, Muller CR, Oldenburg J. Detection of large duplications within the factor VIII gene by MLPA. JThromb Haemost. 2008;6(11):1996-1999. doi: 10.1111/j.1538-7836.2008. 03125.x PMID: 18752578
-
(2008)
JThromb Haemost.
, vol.6
, Issue.11
, pp. 1996-1999
-
-
Rost, S.1
Loffler, S.2
Pavlova, A.3
Muller, C.R.4
Oldenburg, J.5
-
30
-
-
0036212973
-
Aberrant splicing and premature termination of transcription of the FVIII gene as a cause of severe canine hemophilia A: Similarities with the intron 22 inversion mutation in human hemophilia
-
12008949
-
Hough C, Kamisue S, Cameron C, Notley C, Tinlin S, Giles A, et al. Aberrant splicing and premature termination of transcription of the FVIII gene as a cause of severe canine hemophilia A: similarities with the intron 22 inversion mutation in human hemophilia. Thromb Haemost. 2002;87(4):659-665. PMID: 12008949
-
(2002)
Thromb Haemost.
, vol.87
, Issue.4
, pp. 659-665
-
-
Hough, C.1
Kamisue, S.2
Cameron, C.3
Notley, C.4
Tinlin, S.5
Giles, A.6
-
31
-
-
0036790813
-
The Chapel Hill hemophilia A dog colony exhibits a factor VIII gene inversion
-
12242334
-
Lozier JN, Dutra A, Pak E, Zhou N, Zheng Z, Nichols TC, et al. The Chapel Hill hemophilia A dog colony exhibits a factor VIII gene inversion. Proc Natl Acad Sci USA. 2002;99(20):12991-12996. PMID: 12242334
-
(2002)
Proc Natl Acad Sci USA
, vol.99
, Issue.20
, pp. 12991-12996
-
-
Lozier, J.N.1
Dutra, A.2
Pak, E.3
Zhou, N.4
Zheng, Z.5
Nichols, T.C.6
-
32
-
-
84965086065
-
Translational data from adenoassociated virus-mediated gene therapy of hemophilia B in dogs
-
25675273
-
Nichols TC, Whitford MH, Arruda VR, Stedman HH, Kay MA, High KA. Translational data from adenoassociated virus-mediated gene therapy of hemophilia B in dogs. Hum Gene Ther Clin Dev. 2015;26(1):5-14. doi: 10.1089/humc.2014.153 PMID: 25675273
-
(2015)
Hum Gene Ther Clin Dev.
, vol.26
, Issue.1
, pp. 5-14
-
-
Nichols, T.C.1
Whitford, M.H.2
Arruda, V.R.3
Stedman, H.H.4
Kay, M.A.5
High, K.A.6
-
33
-
-
59649122961
-
Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy
-
18957684
-
Niemeyer GP, Herzog RW, Mount J, Arruda VR, Tillson DM, Hathcock J, et al. Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy. Blood. 2009;113(4):797-806. doi: 10.1182/blood-2008-10-181479 PMID: 18957684
-
(2009)
Blood
, vol.113
, Issue.4
, pp. 797-806
-
-
Niemeyer, G.P.1
Herzog, R.W.2
Mount, J.3
Arruda, V.R.4
Tillson, D.M.5
Hathcock, J.6
-
34
-
-
84860155178
-
Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia
-
22185325
-
Demaster A, Luo X, Curtis S, Williams KD, Landau DJ, Drake EJ, et al. Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia. Hum Gene Ther. 2012;23(4):407-418. doi: 10.1089/hum.2011.106 PMID: 22185325
-
(2012)
Hum Gene Ther.
, vol.23
, Issue.4
, pp. 407-418
-
-
Demaster, A.1
Luo, X.2
Curtis, S.3
Williams, K.D.4
Landau, D.J.5
Drake, E.J.6
-
35
-
-
84858005636
-
Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia
-
21697954
-
Li C, Narkbunnam N, Samulski RJ, Asokan A, Hu G, Jacobson LJ, et al. Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia. Gene Ther. 2012;19(3):288-294. doi: 10.1038/gt.2011.90 PMID: 21697954
-
(2012)
Gene Ther.
, vol.19
, Issue.3
, pp. 288-294
-
-
Li, C.1
Narkbunnam, N.2
Samulski, R.J.3
Asokan, A.4
Hu, G.5
Jacobson, L.J.6
-
36
-
-
77954976233
-
Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: Implications for gene therapy using AAV vectors
-
20095819
-
Boutin S, Monteilhet V, Veron P, Leborgne C, Benveniste O, Montus MF, et al. Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors. Hum Gene Ther. 2010;21(6):704-712. doi: 10.1089/hum.2009.182 PMID: 20095819
-
(2010)
Hum Gene Ther.
, vol.21
, Issue.6
, pp. 704-712
-
-
Boutin, S.1
Monteilhet, V.2
Veron, P.3
Leborgne, C.4
Benveniste, O.5
Montus, M.F.6
-
37
-
-
84895532287
-
Humoral Immune Response to AAV
-
24151496
-
Calcedo R, Wilson JM. Humoral Immune Response to AAV. Front Immunol. 2013;4:341. doi: 10.3389/fimmu.2013.00341 PMID: 24151496
-
(2013)
Front Immunol.
, vol.4
, pp. 341
-
-
Calcedo, R.1
Wilson, J.M.2
-
38
-
-
84885107010
-
Humoral immunity to AAV vectors in gene therapy: Challenges and potential solutions
-
23819952
-
Masat E, Pavani G, Mingozzi F. Humoral immunity to AAV vectors in gene therapy: challenges and potential solutions. Discov Med. 2013;15(85):379-389. PMID: 23819952
-
(2013)
Discov Med.
, vol.15
, Issue.85
, pp. 379-389
-
-
Masat, E.1
Pavani, G.2
Mingozzi, F.3
-
39
-
-
84875912760
-
Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue
-
22786533
-
Mingozzi F, Chen Y, Edmonson SC, Zhou S, Thurlings RM, Tak PP, et al. Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue. Gene Ther. 2013;20(4):417-424. doi: 10.1038/gt.2012.55 PMID: 22786533
-
(2013)
Gene Ther.
, vol.20
, Issue.4
, pp. 417-424
-
-
Mingozzi, F.1
Chen, Y.2
Edmonson, S.C.3
Zhou, S.4
Thurlings, R.M.5
Tak, P.P.6
-
40
-
-
84894242894
-
Selection and evaluation of clinically relevant AAV variants in axenograft liver model
-
24390344
-
Lisowski L, Dane AP, Chu K, Zhang Y, Cunningham SC, Wilson EM, et al. Selection and evaluation of clinically relevant AAV variants in axenograft liver model. Nature. 2014;506(7488):382-386. doi: 10. 1038/nature12875 PMID: 24390344
-
(2014)
Nature.
, vol.506
, Issue.7488
, pp. 382-386
-
-
Lisowski, L.1
Dane, A.P.2
Chu, K.3
Zhang, Y.4
Cunningham, S.C.5
Wilson, E.M.6
-
41
-
-
79955597265
-
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins
-
21245849
-
Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, et al. Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther. 2011;19(5):876-885. doi: 10.1038/mt. 2010.274 PMID: 21245849
-
(2011)
Mol Ther.
, vol.19
, Issue.5
, pp. 876-885
-
-
Nathwani, A.C.1
Rosales, C.2
McIntosh, J.3
Rastegarlari, G.4
Nathwani, D.5
Raj, D.6
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