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Search-and-replace genome editing without double-strand breaks or donor DNA
Nature, 2019 Most genetic variants that contribute to disease1 are challenging to correct efficiently and without excess byproducts2–5. Here we describe prime editing, a versatile and precise genome editing method that directly writes new genetic information into a ...
Aditya Raguram +2 more
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Genome editing: the end of the beginning [PDF]
Genome Biology, 2015 Editorial It has recently become commonplace to editorialize on the extent to which genome editing has transformed modern biological research and perhaps, in the future, biomedicine. Nonetheless, each time the scope of scientific progress and the state of the field is appraised, it is followed in rapid succession by another wave of seemingly momentous ...
Jennifer A. Doudna, Charles A. Gersbach
openalex +5 more sources
Scientific Reports, 2022 Recent advances in genome editing technologies have redefined our ability to probe and precisely edit the human genome and epigenome in vitro and in vivo. More specifically, RNA-guided CRISPR/Cas systems have revolutionized the field due to their simplicity in design and adaptability across biological systems.
Maura McGrail +2 more
openaire +5 more sources
Genome Biology, 2022 Advancing crop genomics requires efficient genetic systems enabled by high-quality personalized genome assemblies. Here, we introduce RagTag, a toolset for automating assembly scaffolding and patching, and we establish chromosome-scale reference genomes ...
Michael Alonge +9 more
semanticscholar +1 more source
Genome Editing for Mucopolysaccharidoses [PDF]
International Journal of Molecular Sciences, 2020 Genome editing holds the promise of one-off and potentially curative therapies for many patients with genetic diseases. This is especially true for patients affected by mucopolysaccharidoses as the disease pathophysiology is amenable to correction using multiple approaches. Ex vivo and in vivo genome editing platforms have been tested primarily on MSPI
Edina Poletto +2 more
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Trends in Biotechnology, 2023 A series of spectacular scientific discoveries and technological advances in the second half of the 20th century have provided the basis for the ongoing genome editing revolution. The elucidation of structural and functional features of DNA and RNA was followed by pioneering studies on genome editing: Molecular biotechnology was born.
van der Oost, John +1 more
openaire +2 more sources
N 1-methyl-pseudouridine is incorporated with higher fidelity than pseudouridine in synthetic RNAs
Scientific Reports, 2022 In vitro transcribed synthetic messenger RNAs (mRNAs) represent a novel therapeutic modality. To overcome the inherent immunogenicity, as well as to increase the therapeutic efficacy of the molecules, uridine analogs—such as pseudouridine (Ψ) and N 1 ...
Tien-Hao Chen +4 more
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System analysis of the sequencing quality of human whole exome samples on BGI NGS platform
Scientific Reports, 2022 Human exome sequencing is a classical method used in most medical genetic applications. The leaders in the field are the manufacturers of enrichment kits based on hybridization of cRNA or cDNA biotinylated probes specific for a genomic region of interest.
Vera Belova +10 more
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Deep sampling of gRNA in the human genome and deep-learning-informed prediction of gRNA activities
Cell Discovery, 2023 Life science studies involving clustered regularly interspaced short palindromic repeat (CRISPR) editing generally apply the best-performing guide RNA (gRNA) for a gene of interest.
Heng Zhang +8 more
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Journal of Experimental Medicine, 2023 CRISPR/Cas9 genome editing techniques have the potential to treat previously untreatable inherited genetic disorders of vision by correcting mutations that cause these afflictions. Using a prime editor, Qin et al. (2023. J. Exp. Med.https://doi.org/10.1084/jem.20220776) restored visual functions in a mouse model (rd10) of retinitis pigmentosa.
Samuel W. Du, Krzysztof Palczewski
openaire +3 more sources