Bicistronic AAV Gene Therapy for Tay-Sachs and Sandhoff Diseases

被引：0

作者：

Taghian, Toloo ^{[1
]}

Fernau, Deborah ^{[1
]}

Mercado, Kalajan L. ^{[2
]}

Ellis, Lauren ^{[2
]}

Diffie, Elise ^{[2
]}

Gross, Amanda ^{[2
]}

Maguire, Anne S. ^{[2
]}

Batista, Ana Rita ^{[1
]}

Bertrand, Stephanie ^{[3
]}

Otero, Monique ^{[1
]}

Prestigiacomo, Rachel ^{[3
]}

Gately, Rachael ^{[3
]}

Gallagher, Jillian ^{[1
]}

Lahey, Hannah ^{[1
]}

Taylor, Amanda ^{[2
]}

Koehler, Jey ^{[2
]}

Martin, Douglas R. ^{[2
]}

Sena-Esteves, Miguel ^{[1
]}

Gray-Edwards, Heather L. ^{[1
]}

机构：

[1] Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA USA

[2] Auburn Univ, Auburn, AL 36849 USA

[3] Tufts Cummings Sch Vet Med, Grafton, MA USA

来源：

MOLECULAR THERAPY | 2021年 / 29卷 / 04期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

135

引用

页码：68 / 68

页数：1

共 50 条

[31] MOUSE MODELS OF TAY-SACHS AND SANDHOFF DISEASES DIFFER IN NEUROLOGIC PHENOTYPE AND GANGLIOSIDE METABOLISM
SANGO, K
YAMANAKA, S
HOFFMANN, A
OKUDA, Y
GRINBERG, A
WESTPHAL, H
MCDONALD, MP
CRAWLEY, JN
SANDHOFF, K
SUZUKI, K
PROIA, RL
NATURE GENETICS, 1995, 11 (02) : 170 - 176
[32] Effective Gene Therapy in an Authentic Mouse Model of Tay-Sachs Related Diseases
Cachon-Gonzlez, Begona
Wang, Susan
Ziegler, Robin
Cheng, Seng H.
Cox, Timothy M.
MOLECULAR THERAPY, 2006, 13 : S86 - S87
[33] GENETIC COMPLEMENTATION AFTER FUSION OF TAY-SACHS AND SANDHOFF CELLS
THOMAS, GH
TAYLOR, HA
MILLER, CS
AXELMAN, J
MIGEON, BR
NATURE, 1974, 250 (5467) : 580 - 582
[34] EPIDEMIOLOGY OF THE TAY-SACHS GENE
KABACK, M
AMERICAN JOURNAL OF HUMAN GENETICS, 1981, 33 (06) : A7 - A7
[35] STEM CELL AND GENE THERAPY FOR TAY-SACHS DISEASE
Feinermani, B.
Paino, J. S.
EXPERIMENTAL HEMATOLOGY, 2011, 39 (08) : S98 - S98
[36] A solid start for gene therapy in Tay-Sachs disease
Yu, Timothy W.
Bodamer, Olaf
NATURE MEDICINE, 2022, 28 (02) : 236 - 237
[37] Production of recombinant β-hexosaminidase A, a potential enzyme for replacement therapy for Tay-Sachs and Sandhoff diseases, in the methylotrophic yeast Ogataea minuta
Akeboshi, Hiromi
Chiba, Yasunori
Kasahara, Yoshiko
Takashiba, Minako
Takaoka, Yuki
Ohsawa, Mai
Tajima, Youichi
Kawashima, Ikuo
Tsuji, Daisuke
Itoh, Kohji
Sakuraba, Hitoshi
Jigami, Yoshifumi
APPLIED AND ENVIRONMENTAL MICROBIOLOGY, 2007, 73 (15) : 4805 - 4812
[38] A longitudinal study of hexosaminidase deficiency (Tay-Sachs disease, Sandhoff disease)
Whitley, Chester
Diethelm-Okita, Brenda
Utz, Jeanine
MOLECULAR GENETICS AND METABOLISM, 2013, 108 (02) : S98 - S99
[39] SEGREGATION OF TAY-SACHS AND SANDHOFF ALLELES IN A NON-JEWISH FAMILY
LANE, AB
YOUNG, E
JENKINS, T
AMERICAN JOURNAL OF HUMAN GENETICS, 1980, 32 (06) : 920 - 926
[40] Amelioration of Neurologic Disease after AAV-Gene Therapy in a Sheep Model of Tay-Sachs Disease
Gray-Edwards, H. L.
Hubbard, S. M.
Randle, A. E.
Hwang, M.
Bradbury, A. M.
McCurdy, V. J.
Wilson, D. U.
Hudson, J. A.
Whitlock, B. K.
Sartin, J. L.
Salibi, N.
Beyers, R. J.
Denney, T. S.
Johnson, J. A.
Riddell, K. P.
Cox, N. R.
Sena-Estevez, M.
Martin, D. R.
MOLECULAR THERAPY, 2013, 21 : S17 - S17

← 1 2 3 4 5 →