304 related articles for article (PubMed ID: 32592687)
1. Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice.
Lahey HG; Webber CJ; Golebiowski D; Izzo CM; Horn E; Taghian T; Rodriguez P; Batista AR; Ellis LE; Hwang M; Martin DR; Gray-Edwards H; Sena-Esteves M
Mol Ther; 2020 Oct; 28(10):2150-2160. PubMed ID: 32592687
[TBL] [Abstract][Full Text] [Related]
2. Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease.
Karumuthil-Melethil S; Nagabhushan Kalburgi S; Thompson P; Tropak M; Kaytor MD; Keimel JG; Mark BL; Mahuran D; Walia JS; Gray SJ
Hum Gene Ther; 2016 Jul; 27(7):509-21. PubMed ID: 27197548
[TBL] [Abstract][Full Text] [Related]
3. Reversibility of neuropathology in Tay-Sachs-related diseases.
Cachón-González MB; Wang SZ; Ziegler R; Cheng SH; Cox TM
Hum Mol Genet; 2014 Feb; 23(3):730-48. PubMed ID: 24057669
[TBL] [Abstract][Full Text] [Related]
4. Investigating Immune Responses to the scAAV9-
Kot S; Karumuthil-Melethil S; Woodley E; Zaric V; Thompson P; Chen Z; Lykken E; Keimel JG; Kaemmerer WF; Gray SJ; Walia JS
Int J Mol Sci; 2021 Jun; 22(13):. PubMed ID: 34201771
[TBL] [Abstract][Full Text] [Related]
5. [Molecular pathogenesis and therapeutic approach of GM2 gangliosidosis].
Tsuji D
Yakugaku Zasshi; 2013; 133(2):269-74. PubMed ID: 23370522
[TBL] [Abstract][Full Text] [Related]
6. A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases.
Ou L; Przybilla MJ; Tăbăran AF; Overn P; O'Sullivan MG; Jiang X; Sidhu R; Kell PJ; Ory DS; Whitley CB
Gene Ther; 2020 May; 27(5):226-236. PubMed ID: 31896760
[TBL] [Abstract][Full Text] [Related]
7. Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice.
Osmon KJ; Woodley E; Thompson P; Ong K; Karumuthil-Melethil S; Keimel JG; Mark BL; Mahuran D; Gray SJ; Walia JS
Hum Gene Ther; 2016 Jul; 27(7):497-508. PubMed ID: 27199088
[TBL] [Abstract][Full Text] [Related]
8. Apoptotic cell death in mouse models of GM2 gangliosidosis and observations on human Tay-Sachs and Sandhoff diseases.
Huang JQ; Trasler JM; Igdoura S; Michaud J; Hanal N; Gravel RA
Hum Mol Genet; 1997 Oct; 6(11):1879-85. PubMed ID: 9302266
[TBL] [Abstract][Full Text] [Related]
9. Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates.
Walia JS; Altaleb N; Bello A; Kruck C; LaFave MC; Varshney GK; Burgess SM; Chowdhury B; Hurlbut D; Hemming R; Kobinger GP; Triggs-Raine B
Mol Ther; 2015 Mar; 23(3):414-22. PubMed ID: 25515709
[TBL] [Abstract][Full Text] [Related]
10. Effective gene therapy in an authentic model of Tay-Sachs-related diseases.
Cachón-González MB; Wang SZ; Lynch A; Ziegler R; Cheng SH; Cox TM
Proc Natl Acad Sci U S A; 2006 Jul; 103(27):10373-10378. PubMed ID: 16801539
[TBL] [Abstract][Full Text] [Related]
11. Improvement of motor and behavioral activity in Sandhoff mice transplanted with human CD34+ cells transduced with a HexA/HexB expressing lentiviral vector.
Beegle J; Hendrix K; Maciel H; Nolta JA; Anderson JS
J Gene Med; 2020 Sep; 22(9):e3205. PubMed ID: 32335981
[TBL] [Abstract][Full Text] [Related]
12. Metabolic correction in microglia derived from Sandhoff disease model mice.
Tsuji D; Kuroki A; Ishibashi Y; Itakura T; Itoh K
J Neurochem; 2005 Sep; 94(6):1631-8. PubMed ID: 16092933
[TBL] [Abstract][Full Text] [Related]
13. In cellulo examination of a beta-alpha hybrid construct of beta-hexosaminidase A subunits, reported to interact with the GM2 activator protein and hydrolyze GM2 ganglioside.
Sinici I; Yonekawa S; Tkachyova I; Gray SJ; Samulski RJ; Wakarchuk W; Mark BL; Mahuran DJ
PLoS One; 2013; 8(3):e57908. PubMed ID: 23483939
[TBL] [Abstract][Full Text] [Related]
14. Characterization of inducible models of Tay-Sachs and related disease.
Sargeant TJ; Drage DJ; Wang S; Apostolakis AA; Cox TM; Cachón-González MB
PLoS Genet; 2012 Sep; 8(9):e1002943. PubMed ID: 23028353
[TBL] [Abstract][Full Text] [Related]
15. Inefficiency in GM2 ganglioside elimination by human lysosomal beta-hexosaminidase beta-subunit gene transfer to fibroblastic cell line derived from Sandhoff disease model mice.
Itakura T; Kuroki A; Ishibashi Y; Tsuji D; Kawashita E; Higashine Y; Sakuraba H; Yamanaka S; Itoh K
Biol Pharm Bull; 2006 Aug; 29(8):1564-9. PubMed ID: 16880605
[TBL] [Abstract][Full Text] [Related]
16. Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector.
Osmon KJ; Thompson P; Woodley E; Karumuthil-Melethil S; Heindel C; Keimel JG; Kaemmerer WF; Gray SJ; Walia JS
Curr Gene Ther; 2022; 22(3):262-276. PubMed ID: 34530708
[TBL] [Abstract][Full Text] [Related]
17. Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.
Golebiowski D; van der Bom IMJ; Kwon CS; Miller AD; Petrosky K; Bradbury AM; Maitland S; Kühn AL; Bishop N; Curran E; Silva N; GuhaSarkar D; Westmoreland SV; Martin DR; Gounis MJ; Asaad WF; Sena-Esteves M
Hum Gene Ther; 2017 Jun; 28(6):510-522. PubMed ID: 28132521
[TBL] [Abstract][Full Text] [Related]
18. Biology and potential strategies for the treatment of GM2 gangliosidoses.
Chavany C; Jendoubi M
Mol Med Today; 1998 Apr; 4(4):158-65. PubMed ID: 9572057
[TBL] [Abstract][Full Text] [Related]
19. The biochemistry of HEXA and HEXB gene mutations causing GM2 gangliosidosis.
Mahuran DJ
Biochim Biophys Acta; 1991 Feb; 1096(2):87-94. PubMed ID: 1825792
[No Abstract] [Full Text] [Related]
20. Genetics and Therapies for GM2 Gangliosidosis.
Cachon-Gonzalez MB; Zaccariotto E; Cox TM
Curr Gene Ther; 2018; 18(2):68-89. PubMed ID: 29618308
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
