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Off-target effects of cytidine base editor and adenine base editor: What can we do?
Journal of Genetics and Genomics, 2019Abstract Base editors (BEs) is a powerful tool that could edit the genome efficiently without inducing DNA double-strand breaks (DSBs). Recently, off-target DNA and RNA mutations of BEs were found, rasing concerns about its precision and safety. Here we discuss some strategies that might eliminate off-target effects of BEs.
Puping Liang, Jinkun Wen, Junjiu Huang
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Adenine base editor excels at fixing point mutations
C&EN Global Enterprise, 2017Many diseases are caused by single-base mutations in genomic DNA. Scientists have long searched for methods to correct such mutations to treat these genetic diseases. Last year, David R. Liu of Harvard University and coworkers developed a technique called base editing that fixes some single-base mutations.
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Genome-wide target specificity of CRISPR RNA-guided adenine base editors
Nature Biotechnology, 2019Adenine base editors1 enable efficient targeted adenine-to-guanine single nucleotide conversions to induce or correct point mutations in human cells, animals, and plants1-4. Here we present a modified version of Digenome-seq, an in vitro method for identifying CRISPR (clustered regularly interspaced short palindromic repeats)-induced double-strand ...
Daesik Kim +4 more
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Correcting a patient-specific Rhodopsin mutation with adenine base editor in a mouse model
Molecular TherapyGenome editing offers a great promise to treating human genetic diseases. To assess genome-editing-mediated therapeutic effects in vivo, an animal model is indispensable. The genomic disparities between mice and humans often impede the direct clinical application of genome-editing-mediated treatments using conventional mouse models.
Feng Gu
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Hypercompact adenine base editors based on a Cas12f variant guided by engineered RNA
Nature Chemical Biology, 2022Do Yon Kim +10 more
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A dual-deaminase CRISPR base editor enables concurrent adenine and cytosine editing
Nature Biotechnology, 2020Julian Grünewald +2 more
exaly
Engineered Nme2Cas9 Adenine Base Editors to Treat Rett Syndrome
Rett syndrome (RTT) is a severe X-linked progressive neurodevelopmental disorder that primarily affects females in early childhood. Although the FDA has approved the first drug treatment for RTT, no curative therapy is currently available. Precision genome editing technologies—particularly CRISPR-based adenine base editing (ABE)—offer a promising ...openaire +1 more source