Results 31 to 40 of about 454,543 (260)

Comprehensive assessment of miniature CRISPR-Cas12f nucleases for gene disruption

Nature Communications, 2022
CRISPR-Cas12f nucleases can be effectively packaged into AAVs for gene therapy, but a systematic evaluation of editing outcomes is lacking. Here the authors perform a comprehensive assessment of 4 Cas12f proteins and compare to Cas9 and two Cas12a ...
Changchang Xin, Jianhang Yin, Shaopeng Yuan, Liqiong Ou, Mengzhu Liu, Weiwei Zhang, Jiazhi Hu   +6 more
doaj   +1 more source

Large-Scale CRISPR Screen of LDLR Pathogenic Variants

Research, 2023
Familial hypercholesterolemia (FH) is a frequently occurring genetic disorder that is linked to early-onset cardiovascular disease. If left untreated, patients with this condition can develop severe cardiovascular complications.
Mengjing Li, Lerong Ma, Yiwu Chen, Jianing Li, Yanbing Wang, Wenni You, Hongming Yuan, Xiaochun Tang, Hongsheng Ouyang, Daxin Pang   +9 more
doaj   +1 more source

Phosphatidylinositol 4‐kinase as a target of pathogens—friend or foe?

FEBS Letters, EarlyView.
This graphical summary illustrates the roles of phosphatidylinositol 4‐kinases (PI4Ks). PI4Ks regulate key cellular processes and can be hijacked by pathogens, such as viruses, bacteria and parasites, to support their intracellular replication. Their dual role as essential host enzymes and pathogen cofactors makes them promising drug targets.
Ana C. Mendes, Guilherme M. Azevedo, Amanda P. Barcellos, Diana Bahia   +3 more
wiley   +1 more source

PtWAVE: a high-sensitive deconvolution software of sequencing trace for the detection of large indels in genome editing

BMC Bioinformatics
Background Tracking of Insertions and DEletions (TIDE) analysis, which computationally deconvolves capillary sequencing data derived from the DNA of bulk or clonal cell populations to estimate the efficiency of targeted mutagenesis by programmable ...
Kazuki Nakamae, Saya Ide, Nagaki Ohnuki, Yoshiko Nakagawa, Keisuke Okuhara, Hidemasa Bono   +5 more
doaj   +1 more source

Diversity and complexity in neural organoids

FEBS Letters, EarlyView.
Neural organoid research aims to expand genetic diversity on one side and increase tissue complexity on the other. Chimeroids integrate multiple donor genomes within single organoids. Self‐organising multi‐identity organoids, exogenous cell seeding, or enforced assembly of region‐specific organoids contribute to tissue complexity.
Ilaria Chiaradia, Madeline A. Lancaster
wiley   +1 more source

Methodological landscape in the field of integration site identification of retroviruses and retroviral vectors

Frontiers in Bioengineering and Biotechnology
Detailed mapping of viral vector integration sites, including retroviral and particularly lentiviral vectors, is critical for assessing their safety in preclinical and clinical studies.
Konstantin Kochergin-Nikitsky, Alexander Lavrov, Svetlana Smirnikhina   +2 more
doaj   +1 more source

Differentially activated B cells develop regulatory phenotype and show varying immunosuppressive features: a comparative study

Frontiers in Immunology, 2023
Regulatory B lymphocytes (Bregs) are B cells with well-pronounced immunosuppressive properties, allowing them to suppress the activity of effector cells.
Elina A. Zheremyan, Alina S. Ustiugova, Aksinya N. Uvarova, Nina M. Karamushka, Ekaterina M. Stasevich, Violetta S. Gogoleva, Apollinariya V. Bogolyubova, Nikita A. Mitkin, Dmitry V. Kuprash, Dmitry V. Kuprash, Kirill V. Korneev, Kirill V. Korneev   +11 more
doaj   +1 more source

Hyperosmotic stress induces PARP1‐mediated HPF1‐dependent mono(ADP‐ribosyl)ation

FEBS Letters, EarlyView.
Sorbitol‐induced hyperosmotic stress rapidly induces reversible mono(ADP‐ribosyl)ation (MARylation) on PARP1 without the signs of genotoxic signaling. We show that PARP1 autoMARylation is HPF1 dependent and forms hydroxylamine‐resistant O‐glycosidic linkages.
Anna Georgina Kopasz, Mihály Mérey, Rebeka Vásárhelyi, Ramóna Pék, Victor Imburchia, László Henn, Adrián Kószó, Nicholas D. Lakin, Ivan Ahel, Sébastien Huet, Ágnes Czibula, Gyula Timinszky   +11 more
wiley   +1 more source

Double-strand break-free and transgene-free genome editing in the microalga Nannochloropsis oceanica using removable vectors containing the CRISPR base editing system

Scientific Reports
The accumulation of lipids by algae makes them attractive for carbon-neutral fuel production; however, the industrial-scale production of algal lipids has yet to be achieved.
Keishi Moroi, Takashi Yamamoto, Tomokazu Kurita   +2 more
doaj   +1 more source

Extracellular vesicle-based delivery to airway basal cells for durable gene therapy in cystic fibrosis

Frontiers in Bioengineering and Biotechnology
Airway basal cells are an appealing target for durable genetic therapy in cystic fibrosis because they are long-lived stem/progenitor cells that maintain and regenerate the pseudostratified airway epithelium.
Anastasiia Ivleva, Anna Demchenko, Svetlana Smirnikhina   +2 more
doaj   +1 more source