Bystander editing by adenine base editors impairs vision restoration in a mouse model of Leber congenital amaurosis
Base editors (BEs) have emerged as a powerful tool for gene correction with high activity. However, bystander base editing, a byproduct of BEs, presents challenges for precise editing. Here, we investigated the effects of bystander edits on phenotypic restoration in the context of Leber congenital a...
Saved in:
| Main Authors: | Seok-Hoon Lee, Jun Wu, Dongjoon Im, Gue-ho Hwang, You Kyeong Jeong, Hui Jiang, Seok Jae Lee, Dong Hyun Jo, William A. Goddard, III, Jeong Hun Kim, Sangsu Bae |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2025-06-01
|
| Series: | Molecular Therapy: Methods & Clinical Development |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050125000567 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Similar Items
-
Leber’s Congenital Amaurosis and its Complications – A Very Rare Presentation
by: Sujit Das, et al.
Published: (2024-01-01) -
Leber congenital amaurosis: A clinical and genetic study from a tertiary eye care center
by: Abhishek Upadhyaya, et al.
Published: (2025-05-01) -
A decision making algorithm for inherited retinal dystrophies, caused by biallelic mutations in the RPE65 gene, in the clinical practice of an ophthalmologist
by: E. I. Saidasheva, et al.
Published: (2022-03-01) -
Prospects for the diagnosis and gene therapy of inherited retinal dystrophies caused by biallelic mutations in the RPE65 gene
by: V. V. Neroev, et al.
Published: (2021-09-01) -
Economic outcomes of centralized procurements of gene therapy for patients with orphan diseases: inherited retinal dystrophy
by: N. А. Avxentyev, et al.
Published: (2022-01-01)