Main Monogenic diseases arise from genetic mutations that lead to aberrant or absent gene expression, and many lack appropriate therapies. Advances in molecular biology have enabled several treatment approaches to address this unmet medical need and correct the molecular basis of…

Abstract Diabetic retinopathy is a major cause of vision loss worldwide and areas of retinal non-perfusion (RNP) are a key pathologic feature of the vascular component of diabetic retinopathy. While there is a need for a more complete understanding of the natural history of RNP d…

INTRODUCTION Repurposing bacterial “CRISPR” systems to facilitate RNA-guided cleavage of complementary DNA has revolutionized the field of gene editing in mammalian cells 1 – 4 . S. pyogenes Cas9 has been the most highly used CRISPR system and its use for genome editing only requ…

Introduction Targeted gene editing exploits programmable nucleases, such as zinc finger (ZFN) and RNA-based CRISPR/Cas9 nucleases, to induce a double-stranded break (DSB) into a pre-determined genomic sequence ( Doudna and Charpentier, 2014 , Kim and Kim, 2014 ). Depending on the…

Abstract The β-hemoglobinopathies, including sickle cell disease (SCD) and β-thalassemia, are caused by mutations in the β-globin gene ( HBB ) and affect millions of people worldwide. A curative strategy for the β-hemoglobinopathies would be ex vivo gene correction in patient-der…

Abstract Genome editing by homology directed repair (HDR) is leveraged to precisely modify the genome of therapeutically relevant hematopoietic stem and progenitor cells (HSPCs). Here, we present a new approach to increasing the frequency of HDR in human HSPCs by the delivery of …

Abstract β-Thalassemia pathology is due not only to loss of β-globin ( HBB ), but also to erythrotoxic accumulation and aggregation of the β-globin-binding partner, α-globin ( HBA1/2 ). Here we describe a Cas9/AAV6-mediated genome editing strategy that can replace the entire HBA1…

Ribonucleoprotein (RNP) complexes play central roles in essential cellular processes that include translation, rRNA, pre-mRNA and pre-tRNA processing, and telomere maintenance. In the ribosome and the spliceosome, large RNA molecules form a structural framework for the assembly o…

Reduced Expression of VEGF-A in Human Retinal Pigment Epithelial Cells and Human Muller Cells Following CRISPR-Cas9 Ribonucleoprotein-Mediated Gene Disruption - PMC Skip to main content Official websites use .gov A .gov website belongs to an official government organization in th…

Litchfield County Opiate Task Force | Resource Database Buy Your Tickets to Reimagining Empathy Conference here. NARCAN AVAILABLE HERE Follow us on Facebook Resource Database Find the Help You Need The Task Force is here to help you find the resources you or your loved one needs …

Ancient Egyptian Dictionary konzipiert und kompiliert von Simon D. Schweitzer PDF-Version von simondschweitzer/aed: AED - Ancient Egyptian Dictionary Version 1.0 (Version v1.0) [Data set]. Zenodo. doi.org/10.5281/zenodo.3581069 Dallgow 2020 This work is licensed under a Creative …

Abstract Ex vivo gene correction with CRISPR-Cas9 and a recombinant adeno-associated virus serotype 6 (rAAV6) in autologous hematopoietic stem/progenitor cells (HSPCs) to treat sickle cell disease (SCD) has now entered early-phase clinical investigation. To facilitate the progres…

Abstract CRISPR-Cas-mediated genome editing relies on guide RNAs that direct site-specific DNA cleavage facilitated by the Cas endonuclease. Here we report that chemical alterations to synthesized single guide RNAs (sgRNAs) enhance genome editing efficiency in human primary T cel…

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Analysis of AcMNPV transcriptome using third-generation sequencing In this study, PacBio Sequel and ONT MinION LRS platforms were used to characterize the structure of AcMNPV transcriptome and epitranscriptome (Fig. 1 ). Sequel sequencing yielded a total of 47,880 Circular Consen…