Publications by Lingqiong Cai

Publications by authors named "Lingqiong Cai"

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Treatment of autosomal dominant retinitis pigmentosa caused by RHO-P23H mutation with high-fidelity Cas13X in mice.

Zixiang Yan Yuqin Yao Luyao Li Lingqiong Cai Haiwei Zhang

Mol Ther Nucleic Acids

September 2023

Mutations in (RHO) gene commonly cause autosomal dominant retinitis pigmentosa (adRP) without effective therapeutic treatment so far. Compared with genomic DNA-targeting CRISPR-Cas9 system, Cas13 edits RNA for therapeutic applications, avoiding the risk of causing permanent changes in the genome. In particular, a compact and high-fidelity Cas13X (hfCas13X) recently has been developed to degrade targeted RNA with minimal collateral effects and could also be packaged in a single adeno-associated virus for efficient delivery.

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- Lingqiong Cai's research focuses on developing innovative RNA-targeting therapies for genetic disorders, particularly autosomal dominant retinitis pigmentosa (adRP) caused by mutations in the RHO gene.
- The recent study demonstrates the efficacy of a high-fidelity Cas13X system that edits RNA, offering a safer alternative to traditional CRISPR-Cas9 approaches by minimizing the risk of permanent genomic alterations.
- Findings show that hfCas13X can effectively degrade targeted RNA associated with the RHO-P23H mutation in a mouse model, paving the way for potential therapeutic applications in human conditions.

Publications by authors named "Lingqiong Cai"

Treatment of autosomal dominant retinitis pigmentosa caused by RHO-P23H mutation with high-fidelity Cas13X in mice.

Synopsis of recent research by authors named "Lingqiong Cai"