Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor [PDF]
Molecular Therapy, 2023 : Cystic fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. The 2789+5G>A CFTR mutation is a quite frequent defect causing an aberrant splicing and a non-functional CFTR protein. Here we used
Giulia Maule, Anna Cereseto
exaly +3 more sources
Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity. [PDF]
Nat Biotechnol, 2023 AbstractCytosine base editors (CBEs) are larger and can suffer from higher off-target activity or lower on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that retains the small size, low off-target activity and high on-target activity of current ABEs, we evolved the highly active deoxyadenosine deaminase TadA-8e to
Neugebauer ME +13 more
europepmc +3 more sources
Highly Efficient CRISPR-Mediated Base Editing in Sinorhizobium meliloti [PDF]
Frontiers in Microbiology, 2021 Rhizobia are widespread gram-negative soil bacteria and indispensable symbiotic partners of leguminous plants that facilitate the most highly efficient biological nitrogen fixation in nature.
Longxiang Wang +6 more
doaj +2 more sources
Therapeutic adenine base editing of human hematopoietic stem cells
Nature Communications, 2023 Here, Liao and colleagues apply adenine base editor ABE8e and its PAM-less variant ABE8e-SpRY to β-thalassemia patient hematopoietic stem cells in the form of ribonucleoprotein complexes, resulting in efficient long-term editing and β-thalassemia ...
Jiaoyang Liao +9 more
doaj +2 more sources
CRISPR/Cas9-Mediated Adenine Base Editing in Rice Genome
Rice Science, 2019 SUMMARY: Precise base editing is highly desired in plant functional genomic research and crop molecular breeding. In this study, we constructed a rice-codon optimized adenine base editor (ABE)-nCas9 tool that induced targeted A·T to G·C point mutation of
Li Hao +6 more
doaj +2 more sources
Therapeutic adenine base editor with minimized off-target effects. [PDF]
Protein CellAbstract Genome-wide off-target effect poses a safety risk for clinical use of adenine base editor (ABE), among which ABE8e is one of the most efficient. Genome-wide off-target analysis by two-cell embryo injection (GOTI) analysis showed that the rate of genome-wide single-nucleotide variants (SNVs) in ABE8e-edited cells was ∼30-fold ...
Sun Y +6 more
europepmc +3 more sources
Optimizing plant adenine base editor systems by modifying the transgene selection system [PDF]
Plant Biotechnology Journal, 2020 Ruiying Qin +2 more
exaly +4 more sources
Structure-guided engineering of adenine base editor with minimized RNA off-targeting activity [PDF]
Nature Communications, 2021 Base editors can induce transcriptome-wide RNA off-target editing independent of gRNA. Here, the authors engineer ABEmax variants with minimized RNA off-target activities.
Jianan Li +8 more
doaj +2 more sources
Multiplexed base editing through Cas12a variant-mediated cytosine and adenine base editors
Communications Biology, 2022 Cas12a-mediated cytosine (CBE) and adenine base editor (ABE) systems with elevated efficiencies and expanded target scopes are developed, by combining highly active deaminases with Cas12a variants, enCas12a, RR and RVR, recognizing non-TTTV PAMs.
Fangbing Chen +23 more
doaj +3 more sources
Cytosine base editor 4 but not adenine base editor generates off-target mutations in mouse embryos. [PDF]
Commun Biol, 2020 AbstractDeaminase base editing has emerged as a tool to install or correct point mutations in the genomes of living cells in a wide range of organisms. However, the genome-wide off-target effects introduced by base editors in the mammalian genome have been examined in only one study.
Lee HK +4 more
europepmc +4 more sources