Article

Comprehensive Analysis of CRISPR Base Editing Outcomes for Multimeric Protein

2022-06-20

Abstract excerpt

Point mutations in the KCNJ13 gene cause an autosomal recessive, childhood blindness, Leber congenital amaurosis (LCA16) due to a loss-of-function Kir7.1 channel. In the present study, we investigated the etiology of LCA16 caused by a KCNJ13 missense mutation (c.431T>C, p.Leu144Pro) and explored the activity of two cytosine base editors mRNAs (CBEs, BE4max-WTCas9, and evoCDA-SpCas9-NG) as a proof-of-concept ther...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 9f343855-bbd8-55ef-8203-e0adc9c57d09
DOI: 10.1101/2022.06.20.496792

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Comprehensive Analysis of CRISPR Base Editing Outcomes for Multimeric ProteinDOI 10.1101/2022.06.20.496792Read

Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy.Semantic proximity Read

Nonviral base editing of <i>KCNJ13</i> mutation preserves vision in an inherited retinal channelopathySemantic proximity Read

Genome-wide CRISPR screening identifies cellular factors controlling nonviral genome editing efficiency.Semantic proximity Read

Functional correction of the untreatable CFTR 1717-1G>A mutation through mRNA- and sgRNA-optimized base editing.Semantic proximity Read

Bystander base editing interferes with visual function restoration in Leber congenital amaurosisSemantic proximity Read

Gene augmentation and read-through rescue channelopathy in an iPSC-RPE model of congenital blindnessSemantic proximity Read

Develop a Compact RNA Base Editor by Fusing ADAR with Engineered EcCas6e.Semantic proximity Read

CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin.Semantic proximity Read

Precision A3G base editors for disease modeling and correction.Semantic proximity Read

Engineering single-AAV CRISPR-Cas13 RNA base editors for treatment of inherited retinal diseasesSemantic proximity Read

A Novel KCNJ13 Nonsense Mutation and Loss of Kir7.1 Channel Function Causes Leber Congenital Amaurosis (LCA16).Semantic proximity Read

Allele-specific correction of dominant Best vitelliform macular dystrophy in patient-derived retinal pigment epitheliumSemantic proximity Read