Search Results - "DeWitt, Mark A"
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Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA
Published in Nature biotechnology (01-03-2016)“…The efficiency of homology-directed genome editing with CRISPR-Cas9 is boosted through improved design of donor DNA. Targeted genomic manipulation by Cas9 can…”
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Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
Published in Science translational medicine (12-10-2016)“…Genetic diseases of blood cells are prime candidates for treatment through ex vivo gene editing of CD34 hematopoietic stem/progenitor cells (HSPCs), and a…”
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3
Synthetically modified guide RNA and donor DNA are a versatile platform for CRISPR-Cas9 engineering
Published in eLife (02-05-2017)“…Chemical modification of the gRNA and donor DNA has great potential for improving the gene editing efficiency of Cas9 and Cpf1, but has not been investigated…”
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Cytoplasmic Dynein Moves Through Uncoordinated Stepping of the AAA+ Ring Domains
Published in Science (American Association for the Advancement of Science) (13-01-2012)“…Cytoplasmic dynein is a homodimenc AAA+ motor that transports a multitude of cargos toward the microtubule minus end. How the two catalytic head domains…”
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Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair
Published in Stem cells (Dayton, Ohio) (01-02-2019)“…Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR‐associated system (Cas9)‐mediated gene editing of human hematopoietic stem cells…”
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Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair
Published in Nature biomedical engineering (2017)“…Clustered regularly interspaced short palindromic repeats (CRISPR)–CRISPR associated protein 9 (Cas9)-based therapeutics, especially those that can correct…”
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The AAA3 domain of cytoplasmic dynein acts as a switch to facilitate microtubule release
Published in Nature structural & molecular biology (01-01-2015)“…Cytoplasmic dynein has multiple ATPase subunits, with AAA1 as the primary ATPase. Single-molecule and biochemical approaches reveal that AAA3 ATPase has a role…”
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Distance Mapping in Proteins Using Fluorescence Spectroscopy: The Tryptophan-Induced Quenching (TrIQ) Method
Published in Biochemistry (Easton) (16-11-2010)“…Studying the interplay between protein structure and function remains a daunting task. Especially lacking are methods for measuring structural changes in real…”
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Tension on the linker gates the ATP-dependent release of dynein from microtubules
Published in Nature communications (11-08-2014)“…Cytoplasmic dynein is a dimeric motor that transports intracellular cargoes towards the minus end of microtubules (MTs). In contrast to other processive…”
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Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin
Published in eLife (11-02-2022)“…Naturally occurring point mutations in the promoter switch hemoglobin synthesis from defective adult beta-globin to fetal gamma-globin in sickle cell patients…”
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Bidirectional helical motility of cytoplasmic dynein around microtubules
Published in eLife (28-07-2014)“…Cytoplasmic dynein is a molecular motor responsible for minus-end-directed cargo transport along microtubules (MTs). Dynein motility has previously been…”
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12
ATF4 Regulates MYB to Increase γ-Globin in Response to Loss of β-Globin
Published in Cell reports (Cambridge) (04-08-2020)“…β-Hemoglobinopathies can trigger rapid production of red blood cells in a process known as stress erythropoiesis. Cellular stress prompts differentiating…”
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CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells
Published in PloS one (15-01-2019)“…Sickle Cell Disease and ß-thalassemia, which are caused by defective or deficient adult ß-globin (HBB) respectively, are the most common serious genetic blood…”
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Controlled Cycling and Quiescence Enables Efficient HDR in Engraftment-Enriched Adult Hematopoietic Stem and Progenitor Cells
Published in Cell reports (Cambridge) (01-09-2020)“…Genome editing often takes the form of either error-prone sequence disruption by non-homologous end joining (NHEJ) or sequence replacement by homology-directed…”
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Genome editing via delivery of Cas9 ribonucleoprotein
Published in Methods (San Diego, Calif.) (15-05-2017)“…•CRISPR-Cas genome editing has been employed successfully in many areas of biological research.•Among the remaining challenges in optimizing this platform is…”
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Covalent Protein Labeling and Improved Single-Molecule Optical Properties of Aqueous CdSe/CdS Quantum Dots
Published in ACS nano (25-07-2017)“…Semiconductor quantum dots (QDs) have proven to be superior probes for single-molecule imaging compared to organic or genetically encoded fluorophores, but…”
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High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation
Published in iScience (17-06-2022)“…A point mutation in sickle cell disease (SCD) alters one amino acid in the β-globin subunit of hemoglobin, with resultant anemia and multiorgan damage that…”
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The Conformations of the Manganese Transport Regulator of Bacillus subtilis in its Metal-free State
Published in Journal of molecular biology (02-02-2007)“…The manganese transport regulator (MntR) from Bacillus subtilis binds cognate DNA sequences in response to elevated manganese concentrations. MntR functions as…”
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Antisense targeting of decoy exons can reduce intron retention and increase protein expression in human erythroblasts
Published in RNA (Cambridge) (01-08-2020)“…The decoy exon model has been proposed to regulate a subset of intron retention (IR) events involving predominantly larger introns (>1kb). Splicing reporter…”
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Novel Quantum Dot Probes for Single-Molecule Biophysics
Published in Biophysical journal (28-01-2014)Get full text
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