Tag: ABEs

Future-focused therapeutics | Scientist Live

Base editing is bringing the next breakthrough in cell and gene therapy, explains Michelle Fraser. Cell and gene therapies (CGTs) hold the promise to transform medicine for patients globally. These therapies work by correcting DNA to treat a genetic disease, or by modifying cells from the immune system to specifically…

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Base Editing Strategy for Spinal Muscular Atrophy Rescues Patient Fibroblasts, Reverses Mouse Pathology

A new study describes a base editing method for treating spinal muscular atrophy (SMA). This supports early signs that “single-shot” treatments from clinical trials using genome editing technologies to treat other diseases might work. The study, which was conducted by scientists in the laboratories of Benjamin P. Kleinstiver, PhD, at…

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Precise base editing without unintended indels in human cells and mouse primary myoblasts

Base editors cause unintended indels at the target sites Several types of evolved base editors based on the CRISPR system have been developed for more accurate and efficient genome engineering21,37. Among these, AncBE4max and ABEmax were evolved by modifying codon usage, NLSs, and ancestral deaminase reconstructions21. These modifications greatly improve…

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What Is CRISPR Technology | CitizenSide

History of CRISPR Technology The history of CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology traces back to the early 1990s when scientists first noticed repetitive DNA sequences in the genomes of certain bacteria. However, it wasn’t until 2005 that the true potential of CRISPR was realized. It was in…

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“Transformer” Base Editor Safely and Durably Induces Fetal Hemoglobin in Human HSCs

Though the first CRISPR drug—Casgevy, the CRISPR-Cas9 therapy formerly known as exa-cel developed by Vertex Pharmaceuticals and CRISPR Therapeutics—was approved in the U.K. for the treatment of sickle cell disease, the quest for improved gene editors is far from over. To minimize off-target effects, researchers from Wuhan University tested a…

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IJMS | Free Full-Text | CRISPR/Cas9 Landscape: Current State and Future Perspectives

1. Introduction Genome editing has taken a leading position among genome modification technologies in a short time and is now widely used in gene therapy. To date, there are three main systems for genome editing: zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR/Cas nucleases. Genome editing has…

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Can genome editing transform ocular disease treatment?

In a recent review published in Experimental & Molecular Medicine, researchers reviewed existing data on the discovery and development of gene-editing technologies and their applications in managing ocular diseases. Study: Genome editing in the treatment of ocular diseases. Image Credit: GarnaZarina/Shutterstock.com Background Gene therapy advances offer a new therapeutic approach for various…

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In Vivo Genome Editing of Stem Cells Induced by LNP-based Delivery of mRNA

Part of the job as a pediatric hematologist for Michael P. Triebwasser, MD, PhD, is to take care of patients during their bone marrow transplantation. Just last week, he took care of a patient with a severe combined immunodeficiency (SCID) disorder and another with a bone marrow disorder. Even though…

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HuidaGene Therapeutics creates world’s first guanine base-editor

HuidaGene Therapeutics, a clinical-stage genome-editing company, says the National Science Review has published data from its study of the world’s first DNA base editor converting guanine to cytosine/thymine (pyrimidine), or G-to-Y. The company has filed an international patent application for the glycosylase-based guanine base editor (gGBE) and owns the exclusive…

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HuidaGene Therapeutics Announces Release of World’s First…

2 19.05.2023 – 04:50 Huidagene Therapeutics SHANGHAI and CLINTON, NJ, April 20 /PRNewswire/ HuidaGene Therapeutics (辉大基因; HuidaGene), a clinical-stage genome editing company, announced today that in the online journal National Science Review published comprehensive data from a study on the world‘s first G-to-Y converting DNA base editor, the glycosylase-based guanine…

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HuidaGene Therapeutics Announces Publication of the World’s First Guanine Base-Editor

SHANGHAI and CLINTON, N.J., May 18, 2023 /PRNewswire/ — HuidaGene Therapeutics (辉大基因; HuidaGene), a clinical-stage genome-editing company, today announced the online National Science Review published the comprehensive data from a study of the world’s first DNA base editor converting G-to-Y, glycosylase-based guanine base editor (gGBE). Company has filed an international…

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David Liu Hails Rapid Progress in Precision Genome Editing

David Liu takes center stage at ASGCT. [Kevin Davies] LOS ANGELES – David Liu, PhD, the Harvard University and Broad Institute chemist leading the development of base and prime editing technologies, began his American Society of Gene and Cell Therapy (ASGCT) presidential symposium keynote in humorous vein. That morning, Liu…

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Multiplexed base editing through Cas12a variant-mediated cytosine and adenine base editors

Robust C-to-T conversion by Cas12a variant-mediated CBEs for non-canonical PAMs We initially attempted to screen for optimal cytosine deaminases that confer powerful C-to-T editing for Cas12a-mediated CBEs. Seven dCas12a-CBEs fused with individual cytosine deaminases1,2,16,20,21,22,24,25,26 were compared in HEK293T cells and porcine embryos at sites with TTTV PAM sequences. The dCas12a-A3A…

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Error with file guillaumeKUnitigsAtLeast32bases_all.fasta, kUnitigLengths.txt is of size 0, must be at least of size 1.

Hello, I am trying running an assembly with MaSuRCa but am getting an error at the step: “Computing super reads from PE”. here’s the output with the error: [xxxx@vic Bovidae]$ cd Assembly_test/ [xxxx@vic Assembly_test]$ ls assemble.sh guillaumeKUnitigsAtLeast32bases_all.fasta.tmp masurca_assembly.o4302352 meanAndStdevByPrefix.pe.txt pe_data.tmp quorum_mer_db.jf work1 environment.sh guillaumeKUnitigsAtLeast32bases_all.jump.fasta masurca_config.txt pe.cor.fa pe.renamed.fastq super1.err ESTIMATED_GENOME_SIZE.txt masurca_assembly.e4302352…

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High-purity production and precise editing of DNA base editing ribonucleoproteins

Abstract Ribonucleoprotein (RNP) complex–mediated base editing is expected to be greatly beneficial because of its reduced off-target effects compared to plasmid- or viral vector–mediated gene editing, especially in therapeutic applications. However, production of recombinant cytosine base editors (CBEs) or adenine base editors (ABEs) with ample yield and high purity in…

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