E-LIB Suche - Ergebnisse für: Cheng, Seng H

1

A plain language summary of AAV-based gene therapy: challen..:

Moroz, Tara ; Schulz, Martin ; Levy, Daniel I....
Future Rare Diseases. 4 (2024) 1 - p. , 2024

2

Rationale for using centralized transduction inhibition ass..:

Schulz, Martin ; Bashirians, George ; Cheng, Seng H....
Molecular Therapy - Methods & Clinical Development. 31 (2023) - p. 101119 , 2023

Link: https://doi.org/10.1016/..

3

Binding and neutralizing anti-AAV antibodies: Detection and..:

Schulz, Martin ; Levy, Daniel I. ; Petropoulos, Christos J....
Molecular Therapy. 31 (2023) 3 - p. 616-630 , 2023

Link: https://doi.org/10.1016/..

4

AAV‐mediated expression of galactose‐1‐phosphate uridyltran..:

Brophy, Megan L. ; Stansfield, John C. ; Ahn, Youngwook...
Journal of Inherited Metabolic Disease. 45 (2022) 3 - p. 481-492 , 2022

Link: https://doi.org/10.1002/..

5

Dysregulated DNA methylation in the pathogenesis of Fabry d..:

Shen, Jin-Song ; Balaji, Uthra ; Shigeyasu, Kunitoshi...
Molecular Genetics and Metabolism Reports. 33 (2022) - p. 100919 , 2022

Link: https://doi.org/10.1016/..

6

Glucosylceramide synthase inhibition reduces ganglioside GM..:

Dodge, James C. ; Tamsett, Thomas J. ; Treleaven, Christopher M....
Alzheimer's Research & Therapy. 14 (2022) 1 - p. , 2022

Link: https://doi.org/10.1186/..

7

Substrate reduction therapy using Genz‐667161 reduces level..:

Blumenreich, Shani ; Yaacobi, Chen ; Vardi, Ayelet...
Journal of Neurochemistry. 156 (2020) 5 - p. 692-701 , 2020

Link: https://doi.org/10.1111/..

8

biAb Mediated Restoration of the Linkage between Dystroglyc..:

Gumlaw, Nathan ; Sevigny, Leila M. ; Zhao, Hongmei...
Molecular Therapy. 28 (2020) 2 - p. 664-676 , 2020

Link: https://doi.org/10.1016/..

9

Realizing the promise of gene therapy through collaboration..:

Tretiakova, Anna P. ; Murphy, John E. ; Binks, Michael...
Scientific American. 320 (2019) 2 - p. W1-W5 , 2019

Link: https://www.jstor.org/st..

10

Substrate Reduction Therapy for Sandhoff Disease through In..:

Marshall, John ; Nietupski, Jennifer B. ; Park, Hyejung...
Molecular Therapy. 27 (2019) 8 - p. 1495-1506 , 2019

Link: https://doi.org/10.1016/..

11

Universal Method for the Purification of Recombinant AAV Ve..:

Nass, Shelley A. ; Mattingly, Maryellen A. ; Woodcock, Denise A....
Molecular Therapy - Methods & Clinical Development. 9 (2018) - p. 33-46 , 2018

Link: https://doi.org/10.1016/..

12

Rationally designed AAV2 and AAVrh8R capsids provide improv..:

Sullivan, Jennifer A. ; Stanek, Lisa M. ; Lukason, Michael J....
Gene Therapy. 25 (2018) 3 - p. 205-219 , 2018

Link: https://doi.org/10.1038/..

13

Efficacy of Genz-682452-mediated inhibition of glucosylcera..:

Marshall, John ; Nietupski, Jennifer B. ; Bangari, Dinesh...
Molecular Genetics and Metabolism. 123 (2018) 2 - p. S91-S92 , 2018

Link: https://doi.org/10.1016/..

14

Glucosylceramide synthase inhibition alleviates aberrations..:

Sardi, S. Pablo ; Viel, Catherine ; Clarke, Jennifer...
Proceedings of the National Academy of Sciences of the United States of America. 114 (2017) 10 - p. 2699-2704 , 2017

Link: https://www.jstor.org/st..

15

Priapism in a Fabry disease mouse model is associated with ..:

Meng, Xing‐Li ; Arning, Erland ; Wight‐Carter, Mary...
Journal of Inherited Metabolic Disease. 41 (2017) 2 - p. 231-238 , 2017

Link: https://doi.org/10.1007/..