[HTML][HTML] In vivo somatic cell base editing and prime editing

GA Newby, DR Liu - Molecular Therapy, 2021 - cell.com
Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

Site-directed RNA editing: recent advances and open challenges

HM Khosravi, MF Jantsch - RNA biology, 2021 - Taylor & Francis
ABSTRACT RNA editing by cytosine and adenosine deaminases changes the identity of the
edited bases. While cytosines are converted to uracils, adenines are converted to inosines. If …

A new class of medicines through DNA editing

MH Porteus - New England Journal of Medicine, 2019 - Mass Medical Soc
Clinical Genome Editing Therapeutic editing of the human genome has long been a holy
grail of genetic medicine. Different approaches are founded on a bedrock of basic laboratory …

Prime editing: a new way for genome editing

M Marzec, A Brąszewska-Zalewska, G Hensel - Trends in Cell Biology, 2020 - cell.com
Precise and efficient use of genome editing tools are hampered by the introduction of DNA
double-strand breaks, donor DNA templates, or homology-directed repair. A recent study …

[HTML][HTML] Genome editing: a new approach to human therapeutics

M Porteus - Annual review of pharmacology and toxicology, 2016 - annualreviews.org
The ability to manipulate the genome with precise spatial and nucleotide resolution
(genome editing) has been a powerful research tool. In the past decade, the tools and …

[HTML][HTML] Towards a new era in medicine: therapeutic genome editing

MH Porteus - Genome biology, 2015 - Springer
Genome editing is the process of precisely modifying the nucleotide sequence of the
genome. It has provided a powerful approach to research questions but, with the …

[HTML][HTML] Base and prime editing technologies for blood disorders

P Antoniou, A Miccio, M Brusson - Frontiers in genome editing, 2021 - frontiersin.org
Nuclease-based genome editing strategies hold great promise for the treatment of blood
disorders. However, a major drawback of these approaches is the generation of potentially …

Effective gene editing by high-fidelity base editor 2 in mouse zygotes

P Liang, H Sun, Y Sun, X Zhang, X Xie, J Zhang… - Protein & …, 2017 - academic.oup.com
Targeted point mutagenesis through homologous recombination has been widely used in
genetic studies and holds considerable promise for repairing disease-causing mutations in …

Analysis and minimization of cellular RNA editing by DNA adenine base editors

HA Rees, C Wilson, JL Doman, DR Liu - Science advances, 2019 - science.org
Adenine base editors (ABEs) enable precise and efficient conversion of target A• T base
pairs to G• C base pairs in genomic DNA with a minimum of by-products. While ABEs have …

Base editors for simultaneous introduction of C-to-T and A-to-G mutations

RC Sakata, S Ishiguro, H Mori, M Tanaka… - Nature …, 2020 - nature.com
We describe base editors that combine both cytosine and adenine base-editing functions. A
codon-optimized fusion of the cytosine deaminase PmCDA1, the adenosine deaminase …