Results 171 to 180 of about 46,126 (304)

Determination of gRNA Efficiency In Vivo v1

open access: yes
Dissecting how specific genes causally contribute to the function of defined cell types requires knocking them out in vivo. While CRISPR/Cas9 has revolutionized gene editing and genomic engineering, methods to characterize its editing efficiency after in vivo expression have been lacking. This protocol addresses that gap by quantifying guide RNA (gRNA)
Carl V. L. Olson   +3 more
openaire   +1 more source

Atrial natriuretic peptide counteracts aldosterone secretion by preventing acute angiotensin II‐induced cAMP signalling

open access: yesBritish Journal of Pharmacology, EarlyView.
Background and Purpose Aldosterone plays a key role in blood pressure and volume regulation. Enhanced aldosterone secretion contributes to cardiovascular and renal diseases. Therefore, inhibitors of aldosterone receptors and synthase are essential drugs for the treatment of hypertension and heart failure.
Sanika Mohagaonkar   +6 more
wiley   +1 more source

Repurposed catechol‐O‐methyltransferase inhibitors tolcapone and entacapone preferentially suppress aggregation and seeding of P301 mutant tau in human neuronal models

open access: yesBritish Journal of Pharmacology, EarlyView.
Background and purpose Pathogenic aggregation and propagation of seed‐competent TAU assemblies drive tauopathies. MAPT P301 mutations accelerate aggregation and enhance seed competence, yet pharmacological strategies selectively targeting these pathogenic species remain limited.
Ihor Kozlov   +6 more
wiley   +1 more source

Genetic Engineering of Tumor‐Infiltrating Lymphocytes (TIL) via a T‐Editor Platform to Enhance Anti‐Tumor Activity

open access: yesCancer Science, EarlyView.
A rapid and efficient CRISPR‐mediated gene editing platform for TIL engineering identified FAM84B as a novel potential target to enhance antitumor activity and pioneered the use of CBE to generate FAM84B loss‐of‐function TIL with enhanced antitumor activity.
Fenge Li   +13 more
wiley   +1 more source

CRISPRware: an efficient method for contextual gRNA library design

open access: yes
Abstract We present CRISPRware, an efficient method for generating guide RNA (gRNA) libraries against transcribed, translated, and noncoding regions. CRISPRware leverages next-generation sequencing data to design context-specific gRNAs and accounts for genetic variation, which allows allele-specific guide design on a genome-wide scale ...
Eric Malekos   +2 more
openaire   +2 more sources

Challenges in Gene‐Directed Therapies for Rare Neurodevelopmental Disorders Using HNRNPU as an Exemplar

open access: yesClinical Genetics, EarlyView.
Beyond diagnosing rare neurodevelopmental disorders like HNRNPU‐RNDD, developing gene‐directed therapies requires overcoming complex hurdles, including spatial, developmental, and cell‐type constraints. Addressing these requires understanding the molecular effects of dosage and chromatin dynamics to deliver precise, effective treatments.
Daniel Aiyedun   +3 more
wiley   +1 more source

Ploidy‐Dependent Response to Anticancer Drugs of Human Embryonic Stem Cells

open access: yesCell Proliferation, EarlyView.
Using isogenic hESCs, differing only in their ploidy level, we show the effect of ploidy on the resistance to anticancer drugs. We demonstrate that polyploidy, by itself, confers sensitivity to chemotherapies, leading to higher apoptosis and delayed proliferation, while the loss of p53 reverses the phenotype, demonstrating higher resistance.
Guy Haim‐Abadi, Nissim Benvenisty
wiley   +1 more source

Derivation of Mouse Dome‐Shaped Trophoblast Stem Cells With Low Heterogeneity From Blastocysts

open access: yesCell Proliferation, EarlyView.
Here, Liu et al. report the generation of mouse dome‐shaped trophoblast stem cells (dTSCs) with low heterogeneity and autonomous differentiation potential. dTSCs robustly generate all trophoblast lineages in vitro and contribute to the placenta in vivo, providing a refined model for studying trophoblast development and related disorders.
Minli Liu   +8 more
wiley   +1 more source

BCL2 Facilitates Naïve Pluripotency and an 8C‐Like State in Human Embryonic Stem Cells

open access: yesCell Proliferation, EarlyView.
BCL2 stabilizes human naïve pluripotency associated with H3K27ac modulation and induces 8CLCs in human naïve ESCs. ABSTRACT Human naïve embryonic stem cells (ESCs) can spontaneously transition into 8‐cell‐like cells (8CLCs), a cell state that recapitulates the totipotent features of the 8‐cell‐stage embryo.
Zhimin Lu   +7 more
wiley   +1 more source

Transgenic selection and underlying mechanisms in apicomplexan parasites

open access: yesThe FEBS Journal, EarlyView.
Transgenic selection markers have driven genetic engineering in apicomplexans, enabling precise, iterative experiments. This review discusses mechanistic details of drug selection markers, strategies for marker recycling, and practical considerations for several clinically relevant parasites.
Swaroop Peddiraju   +2 more
wiley   +1 more source

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