Precise progerin targeting using RfxCas13d: A therapeutic avenue for Hutchinson-Gilford progeria syndrome.

doi:10.1016/j.ymthe.2025.06.017

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Article

Precise progerin targeting using RfxCas13d: A therapeutic avenue for Hutchinson-Gilford progeria syndrome.

Molecular therapy : the journal of the American Society of Gene Therapy - 3 Sept 2025

Chae Unbin, Yang Hae-Jun, Kim Hanseop, Lee Seung Hwan, Lee Dong Gil, Koo Jeong Young, Ha Seung-Min, Bak Seo-Jong, Joo Mina, Nam Hyun Hee, Lim Kyung-Seob, Kang Philyong, Son Hee-Chang, An You Jeong, Kim Young-Hyun, Song In-Sung, Lee Sang-Hee, Kim Hae Rim, Cho Sang-Mi, Kim Eun-Kyoung, Nam Ki-Hoan, Chung Kyung-Sook, Kim Jae-Yoon, Kim Seon-Yeop, Kim Seon-Kyu, Kim Seon-Young, Lee Dong-Seok, Kim Jin-Man, Park Young-Ho, Kim Sun-Uk

Abstract excerpt

Hutchinson-Gilford progeria syndrome (HGPS), an extremely rare progressive genetic disorder, is caused by a point mutation in LMNA that induces progerin production, which disrupts cellular function and triggers premature aging and mortality. Despite extensive efforts, HPGS remains incurable. We successfully implemented a strategy using RfxCas13d to selectively target progerin mRNA at specific junction regions,...

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Topics

Progeria
Animals
Mice
Humans

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Lamin Type A

Disease Models, Animal

RNA, Messenger

Regulatory Factor X Transcription Factors

Phenotype

RNA Splicing

DNA Damage

Source

Open in PubMed Open full text

Identifiers

PMID: 40518667
DOI: 10.1016/j.ymthe.2025.06.017
PMCID: PMC12432902

Genes

LMNA

Variants

Provenance

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