Zobrazeno 1 - 2
of 2
pro vyhledávání: '"Sydney Lehman"'
Autor:
Yi A. Chen, Mark W. Kankel, Sam Hana, Shukkwan Kelly Lau, Maria I. Zavodszky, Olivia McKissick, Nicole Mastrangelo, Jessica Dion, Bin Wang, Daniel Ferretti, David Koske, Sydney Lehman, Kathryn Koszka, Helen McLaughlin, Mei Liu, Eric Marshall, Attila J. Fabian, Patrick Cullen, Galina Marsh, Stefan Hamann, Michael Craft, Jennifer Sebalusky, H. Moore Arnold, Rachelle Driscoll, Adam Sheehy, Yi Luo, Sonia Manca, Thomas Carlile, Chao Sun, Kirsten Sigrist, Alexander McCampbell, Christopher E. Henderson, Shih-Ching Lo
Publikováno v:
Gene Therapy. 30:443-454
CRISPR-based gene editing technology represents a promising approach to deliver therapies for inherited disorders, including amyotrophic lateral sclerosis (ALS). Toxic gain-of-function superoxide dismutase 1 (SOD1) mutations are responsible for ~20%
Autor:
Nicole Mastrangelo, Tess Torregrosa, Kathryn Koszka, Alexander McCampbell, Shih-Ching Lo, Sydney Lehman, Shanqin Xu, Christopher E Henderson, Sam Hana, Galina Marsh
Publikováno v:
Gene Therapy
Adeno-associated virus (AAV) transduction efficiency and tropism are conventionally determined by high expression of a fluorescent reporter gene. Emerging data has suggested that such conventional methods may underestimate AAV transduction for cells