Base editing: advances and therapeutic opportunities

EM Porto, AC Komor, IM Slaymaker… - Nature Reviews Drug …, 2020 - nature.com
… in living cells — is one of the most recent advances in the field of genome … cells process
base editing intermediates may additionally inform new therapeutic strategies using base editors

Therapeutic base editing of human hematopoietic stem cells

J Zeng, Y Wu, C Ren, J Bonanno, AH Shen, D Shea… - Nature Medicine, 2020 - nature.com
Live cell images were acquired using a Nikon Eclipse Ti inverted microscope. Image
acquisition was performed at room temperature and air in a 24-well plate. Sickled …

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

GA Newby, JS Yen, KJ Woodard, T Mayuranathan… - Nature, 2021 - nature.com
… Adenine base editors (ABEs) convert targeted A•T base pairs to G•C in living cells without
requiring DSBs or donor DNA templates, and with minimal formation of indels 2 . In SCD, the …

CRISPR/Cas-mediated base editing: technical considerations and practical applications

KA Molla, Y Yang - Trends in biotechnology, 2019 - cell.com
… Because new base editors significantly reduce unintended editing in the … base editors,
assess their technical advantages and limitations, and discuss their broad applications in basic

Precise plant genome editing using base editors and prime editors

KA Molla, S Sretenovic, KC Bansal, Y Qi - Nature Plants, 2021 - nature.com
Base editing Base editing is a new technology for the precise modification of genomes (DNA)
or transcriptomes (RNA) of living cells at the single-base resolution. BEs are composed of …

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

JM Levy, WH Yeh, N Pendse, JR Davis… - Nature biomedical …, 2020 - nature.com
… of point mutations in living cells, we developed base editors—… deliver base editors using
AAVs, we devised a split base-editor … Each base-editor half is fused to half of a fast-splicing split …

CRISPR DNA base editors with reduced RNA off-target and self-editing activities

J Grünewald, R Zhou, S Iyer, CA Lareau… - Nature …, 2019 - nature.com
… off-target RNA-editing activity of DNA base editors, expands the options available to minimize
these unwanted effects, and provides new SECURE base editor architectures with other …

Determinants of base editing outcomes from target library analysis and machine learning

M Arbab, MW Shen, B Mok, C Wilson, Ż Matuszek… - Cell, 2020 - cell.com
… do not fully capture the scope of base editing. A systematic and comprehensive … of base
editing would enhance our understanding of base editors, facilitate their use in precision editing

Treatment of a mouse model of ALS by in vivo base editing

CKW Lim, M Gapinske, AK Brooks, WS Woods… - Molecular Therapy, 2020 - cell.com
… Although ALS is currently incurable, CRISPR base editors hold … in vivo delivery of cytidine
base editors (CBEs) consisting of the … This work expands the capabilities of single-base editors

Targeted A-to-G base editing in human mitochondrial DNA with programmable deaminases

SI Cho, S Lee, YG Mok, K Lim, J Lee, JM Lee, E Chung… - Cell, 2022 - cell.com
… In an effort to create adenine base editors enabling A-to-G substitutions in organelle DNA,
we first investigated whether fusion proteins containing a TALE protein custom designed to …