Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

By Morgan L. Maeder, Michael Stefanidakis, Christopher J. Wilson, Reshica Baral, Luis A. Barrera, G. Bounoutas, D. Bumcrot, Hoson Chao, Dawn M. Ciulla, Jen DaSilva and 27 more. Nature Medicine, 2019

610 citations recorded by the source corpus.

Subfields: Molecular Biology

View Paper: https://www.semanticscholar.org/paper/7ca786348bc41c4a61023549244afe46cb423554

How researchers see this paper

The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

In Molecular Biology

1. Joy Y. Wang
2. Min Wu
3. Mengyuan Huang
4. Hongyi Li
5. Xia Zhao
6. Yang Yang
7. Guangping Gao
8. Wei Zhan

Citations in the corpus

• CRISPR technology: A decade of genome editing is only the beginning (2023, background): “However, direct injection results in genome editing of relatively few cells within a localized space and is practical only for organs directly accessible to such injection (161, 169).”
• Adeno-associated virus as a delivery vector for gene therapy of human diseases (2024, background): “Examples include therapeutic transgenes like centrosomal protein ( CEP290 ) for Leber ’ s congenital amaurosis type 10 (~7.5 kb), 145 or CRISPR-based tools like cytosine base editor or prime editor 2 (>5 kb).”
• CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future (2020, background): “The DSB induced by Cas9 and both guides result in either a deletion or inversion of the IVS26 intronic region, thus preventing the aberrant splicing caused by the genetic mutation and enabling subsequent translation of the functional protein (107).”
• Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects (2020, positive): “These extraordinary studies provide a roadmap for the preclinical advance of gene therapy for LCA10.”