Safety and long-term efficacy of AAV2-BEST1 gene augmentation therapy in canine model of Best vitelliform macular dystrophies

被引：0

作者：

Guziewicz, Karina E. ^{[1
]}

Cideciyan, Artur V. ^{[2
]}

Kendrick, Brian T. ^{[1
]}

Dufour, Valerie L. ^{[1
]}

Ruthel, Gordon ^{[3
]}

Komaromy, Andras M. ^{[4
]}

Iwabe, Simone ^{[1
]}

Hauswirth, William ^{[5
]}

Beltran, William A. ^{[1
]}

Jacobson, Samuel G. ^{[2
]}

Aguirre, Gustavo D. ^{[1
]}

机构：

[1] Univ Penn, Clin Sci & Adv Med, Philadelphia, PA 19104 USA

[2] Univ Penn, Dept Ophthalmol, Philadelphia, PA 19104 USA

[3] Univ Penn, Dept Pathobiol, Philadelphia, PA 19104 USA

[4] Michigan State Univ, Dept Small Anim Clin Sci, E Lansing, MI 48824 USA

[5] Univ Florida, Dept Ophthalmol, Gainesville, FL 32611 USA

来源：

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE | 2019年 / 60卷 / 09期

关键词：

D O I：

暂无

中图分类号：

R77 [眼科学];

学科分类号：

100212 ;

摘要：

5200

引用

页数：3

共 50 条

[21] Long-term safety and efficacy of stem cell gene therapy for ADA-SCID.
Aiuti, Alessandro
Benninghoft, Ulrike
Cassani, Barbara
Cattaneo, Federica
Callegaro, Luciano
Andolfi, Grazia
Mirolo, Massimiliano
Scaramuzza, Samantha
Marktel, Sarah
Tabucchi, Antonella
Carlucci, Filippo
Eibl, Martha
Aker, Memet
Slavin, Shimon
Ciceri, Fabio
Miniero, Roberto
Bordignon, Claudio
Roncarolo, Maria Grazia
BLOOD, 2006, 108 (11) : 63A - 63A
[22] Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
Gaudet, D.
Methot, J.
Dery, S.
Brisson, D.
Essiembre, C.
Tremblay, G.
Tremblay, K.
de Wal, J.
Twisk, J.
van den Bulk, N.
Sier-Ferreira, V.
van Deventer, S.
GENE THERAPY, 2013, 20 (04) : 361 - 369
[23] Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
D Gaudet
J Méthot
S Déry
D Brisson
C Essiembre
G Tremblay
K Tremblay
J de Wal
J Twisk
N van den Bulk
V Sier-Ferreira
S van Deventer
Gene Therapy, 2013, 20 : 361 - 369
[24] LONG-TERM CORRECTION OF ARGINASE DEFICIENCY IN A MURINE MODEL USING AAV-BASED GENE THERAPY
Kosenblatt, R. A.
Lay, F. D.
Cederbaum, S. D.
Grody, W. W.
Lipshutz, G. S.
MOLECULAR GENETICS AND METABOLISM, 2009, 98 (1-2) : 143 - 143
[25] Long-term efficacy of AAV-Npc2 gene therapy for severe Niemann-Pick Type C2 disease
Marco, S.
Sanchez, X.
Elias, G.
Ribera, A.
Sanchez, V.
Bertolin, J.
Perez, J.
Roca, C.
Garcia, M.
Molas, M.
Leon, X.
Ruberte, J.
Storch, J.
Bosch, F.
HUMAN GENE THERAPY, 2024, 35 (3-4) : A181 - A181
[26] Long-term functional rescue following a AAV-based gene therapy in canine model of LRIT3-congenital stationary night blindness
Takahashi, Kei
Kwok, Jennifer C.
Sato, Yu
Aguirre, Gustavo D.
Miyadera, Keiko
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2023, 64 (08)
[27] Long-Term Safety and Efficacy of Retroviral-Mediated Gene Therapy for ADA-SCID
Cicalese, M. P.
Ferrua, F.
Pajno, R.
Barzaghi, F.
Frittoli, M.
Castagnaro, L.
Giannelli, S.
Dionisio, F.
Brigida, I.
Tabucchi, A.
Carlucci, F.
Hogg, A.
Singh, K.
Campanile, A.
Philipson, R.
Appleby, J.
Roncarolo, M. G.
Aiuti, A.
JOURNAL OF CLINICAL IMMUNOLOGY, 2014, 34 : S311 - S311
[28] Long-Term Efficacy and Safety in Preclinical Hematopoietic Stem Cell Gene Therapy in Pompe Mice
van Til, Niek P.
Yoon, John K.
Schindler, Rena
Dogan, Yildirim
Barese, Cecilia
Unnisa, Zeenath
Jacobs, Mary
Pizzurro, Daniella
Oborski, Christine
Rothe, Michael
Schambach, Axel
Yan, Aimin
Biasco, Luca
Pfeifer, Richard
Mason, Chris
MOLECULAR THERAPY, 2022, 30 (04) : 492 - 493
[29] Long-term therapy of PKU in mice by AAV2 pseudotype 8 gene transfer to liver
Thony, B.
Rebuffat, A.
Georgiev, P.
Ding, Z.
JOURNAL OF INHERITED METABOLIC DISEASE, 2006, 29 : 93 - 93
[30] Long-term efficacy of a single administration of AAV lipoprotein lipase gene therapy corrects murine hypertriglyceridemia and raises HDL
Ross, CJD
Kuivenhoven, JA
Liu, GQG
Miao, F
Scheenhart-van der Meer, JWC
Moraal, E
Oranje, PPA
Hermens, WTJMC
Kastelein, JJP
Hayden, MR
Meulenberg, JJM
CIRCULATION, 2002, 106 (19) : 126 - 126

← 1 2 3 4 5 →