Sangsu Bae

In vivo base editing rescues hearing in humanized MPZL2 mice within a structure-dependent therapeutic window

Sang‐Yeon Lee, Sangsu Bae, Sohyang Jeong, Byeong Hee Kang et autres

kr, Éthiopie, us (code pays fourni par la source)

Highly efficient base editing at PCSK9 and normal human embryo development

Stepan Jerabek, Chanju Jung, Michelle Kappy, Qiaojin Zhao et autres

cz, us, kr (code pays fourni par la source)

Optimized Cas9‐Enriched Nanopore Sequencing and Analysis Workflow for Clinical Diagnosis of Repeat Expansion Disorders

Seungbok Lee, Chanju Jung, Minjeong Kim, Narae Kim et autres

Short tandem repeat (STR) expansion is a major genetic mechanism underlying numerous neurogenetic disorders. However, traditional PCR amplification and short-read next-generation sequencing-based methods often fail to detect large-scale, complex expansions and to capture methylation information. Thus, this study aimed to modify an …

kr, us, am (code pays fourni par la source)

Precise genomic integration of large DNA fragments by donor-directed annealing using prime editing

Hojun Jung, Bada Jeong, Yong-Woo Kim, Chanju Jung et autres

Replacing large-scale fragments in human cells remains a substantial challenge. Here, we present a programmable gene replacement tool, named prime assembly (PA), which adapts prime editors to produce one or two pairs of 3'-flaps on both the genome and donor DNA. These …

kr, us, pr, am (code pays fourni par la source)

Engineered ADARs enable precision A-to-G base editing of DNA

Hyeon Woo Im, Bada Jeong, Yeji Lee, Ye Eun Oh et autres

Adenine base editors (ABEs), which enable A•T-to-G•C base editing, have emerged as a powerful tool with potential therapeutic applications. However, conventional ABEs suffer from bystander nucleotide conversions, limiting their utility for precise editing. Here we present a single-nucleotide resolution ABE (snuABE) created …

kr, us, pr, am (code pays fourni par la source)

Topical application of Cas9 ribonucleoproteins inhibits corneal neovascularization in a mouse model of alkali burn injury

Seok Jae Lee, Bae-Geun Nam, Sung-Ah Hong, Dong Hyun Jo et autres

kr, pr, Soudan (code pays fourni par la source)

Neuronal mitochondrial disaggregase CLPB ameliorates Huntington's disease pathology in mice

Hyeonho Kim, Gaeun Hyun, Seunghye Kim, Chi-Ho Yu et autres

Background: Huntington's disease (HD) is a devastating neurodegenerative disorder caused by CAG repeat expansion in the HTT gene, resulting in a polyglutamine-expanded huntingtin (HTT) protein that forms toxic aggregates.Although heat-shock proteins are known to facilitate the refolding or clearance of misfolded proteins, …

kr, Éthiopie, us (code pays fourni par la source)

Cas9-enriched nanopore sequencing enables comprehensive and multiplexed detection of repeat expansions

Seungbok Lee, Chan Y. Jung, Min-Jeong Kim, Na Rae Kim et autres

Abstract Short tandem repeat (STR) expansion is a major genetic mechanism underlying numerous neurogenetic disorders. However, traditional PCR amplification and short-read next-generation sequencing (NGS)-based methods often fail to detect complex and large expansions including methylation information. Here, we modified an amplification-free nanopore …

kr, Éthiopie (code pays fourni par la source)

Expansion of artificial intelligence for genome editing

Heesoo Uhm, Sangsu Bae

kr, us, pr, am (code pays fourni par la source)

TMOD-01. Prime Editing of ATRX point mutations in glioblastoma reveals lethal dependencies and targetable vulnerabilities

Ye Ji Lee, Emily Outwin, Timothy C. Humphrey, Sangsu Bae

Abstract Glioblastoma multiforme (GBM) is a highly aggressive and treatment-resistant brain tumour. ATRX mutations are frequently observed in both adult and paediatric GBM, including many single-residue missense mutations. These remain underexplored due to technical limitations in modelling, as conventional knockout models do …

kr, gb (code pays fourni par la source)

Site-specific replacement of large-scale DNA fragments in human cells

Yong-Woo Kim, Chan Y. Jung, Heesoo Uhm, Hyoungrak Kim et autres

Abstract Despite advances in genome editing 1-4 , precisely replacing large-scale fragments in human cells remains a significant challenge. Here, we present a site-specific gene replacement tool, named Prime Assembly (PA), which adapts prime editors to produce one or two pairs of …

kr, Éthiopie (code pays fourni par la source)

ER Chaperone HYOU1 Couples MAM Remodeling to Autophagic Lipid Clearance in Atherosclerosis

Ho Jeong Kwon, Minjeong Ko, Sunyoung Kwon, Hye Eun Lee et autres

kr, Éthiopie (code pays fourni par la source)