Pre-clinical validation of AAV-mediated gene therapy for KCNV2 retinopathy improves visual function and expression in mouse and patient models

Rabab Rashwan, Paula I. Fuller-Carter, Xin Ru Lim, Alicia A. Brunet, Annie L. Miller, Yashvi Bhatt, Rebekah James, Denise Anderson, Valentina Voigt, Joao A. Paulo, Mehdi Mirzaei, Melissa M. Mangala, Emilie O. Wong, Robyn V. Jamieson, Anai Gonzalez-Cordero, David M. Hunt, Livia S. Carvalho

Open source

DOI
10.1016/j.ymthe.2026.09.025
Published
2026-09
Container
Molecular Therapy
Publisher
Elsevier BV
Open access
unknown

Credibility signals

uncertain Score 64/100 under policy 1.0.0. This is a metadata assessment, not a judgment of the paper's conclusions.

Show all credibility signals

Cite this work

BibTeX

@article{allodium:10.1016/j.ymthe.2026.09.025,
  title = {Pre-clinical validation of AAV-mediated gene therapy for KCNV2 retinopathy improves visual function and expression in mouse and patient models},
  author = {Rabab Rashwan and Paula I. Fuller-Carter and Xin Ru Lim and Alicia A. Brunet and Annie L. Miller and Yashvi Bhatt and Rebekah James and Denise Anderson and Valentina Voigt and Joao A. Paulo and Mehdi Mirzaei and Melissa M. Mangala and Emilie O. Wong and Robyn V. Jamieson and Anai Gonzalez-Cordero and David M. Hunt and Livia S. Carvalho},
  year = {2026},
  journal = {Molecular Therapy},
  doi = {10.1016/j.ymthe.2026.09.025},
  url = {https://doi.org/10.1016/j.ymthe.2026.09.025}
}

RIS

TY  - JOUR
TI  - Pre-clinical validation of AAV-mediated gene therapy for KCNV2 retinopathy improves visual function and expression in mouse and patient models
AU  - Rabab Rashwan
AU  - Paula I. Fuller-Carter
AU  - Xin Ru Lim
AU  - Alicia A. Brunet
AU  - Annie L. Miller
AU  - Yashvi Bhatt
AU  - Rebekah James
AU  - Denise Anderson
AU  - Valentina Voigt
AU  - Joao A. Paulo
AU  - Mehdi Mirzaei
AU  - Melissa M. Mangala
AU  - Emilie O. Wong
AU  - Robyn V. Jamieson
AU  - Anai Gonzalez-Cordero
AU  - David M. Hunt
AU  - Livia S. Carvalho
PY  - 2026
JO  - Molecular Therapy
DO  - 10.1016/j.ymthe.2026.09.025
UR  - https://doi.org/10.1016/j.ymthe.2026.09.025
ER  - 

APA

Rashwan, R., Fuller-Carter, P. I., Lim, X. R., Brunet, A. A., Miller, A. L., Bhatt, Y., James, R., Anderson, D., Voigt, V., Paulo, J. A., Mirzaei, M., Mangala, M. M., Wong, E. O., Jamieson, R. V., Gonzalez-Cordero, A., Hunt, D. M., & Carvalho, L. S. (2026). Pre-clinical validation of AAV-mediated gene therapy for KCNV2 retinopathy improves visual function and expression in mouse and patient models. Molecular Therapy. https://doi.org/10.1016/j.ymthe.2026.09.025

Source records