Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa

Occelli, L. M., Zobel, L., Stoddard, J., Wagner, J., Pasmanter, N., Querubin, J., Renner, L. M., Reynaga, R., Winkler, P. A., Sun, K., Marinho, L. F. L. P., O’Riordan, C. R., Frederick, A., Lauer, A., Tsang, S. H., Hauswirth, W. W., McGill, T. J., Neuringer, M., Michalakis, S., & Petersen-Jones, S. M. (2023). Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa. Molecular Therapy, 31(7), 2028–2041. https://doi.org/10.1016/j.ymthe.2023.04.005
Authors:
Laurence M. Occelli
Lena Zobel
Jonathan Stoddard
Johanna Wagner
Nathaniel Pasmanter
Janice Querubin
Lauren M. Renner
Rene Reynaga
Paige A. Winkler
Kelian Sun
Luis Felipe L.P. Marinho
Catherine R. O'Riordan
Amy Frederick
Andreas Lauer
Stephen H. Tsang
William W. Hauswirth
Trevor J. McGill
Martha Neuringer
Stylianos Michalakis
Simon M. Petersen-Jones
Affiliated Authors:
Stephen H. Tsang
Author Keywords:
adeno-associated virus
cngb1
dog
electroretinography
gene therapy
nonhuman primate
perifoveal chorioretinal atrophy
retinitis pigmentosa
short rhodopsin promoter
spectral domain optical coherence tomography
Publication Type:
Article
Unique ID:
10.1016/j.ymthe.2023.04.005
PMID:
Publication Date:
Data Source:
Scopus

Record Created: