
Cell stem cell, Год журнала: 2023, Номер 30(11), С. 1452 - 1471.e10
Опубликована: Окт. 12, 2023
Язык: Английский
Cell stem cell, Год журнала: 2023, Номер 30(11), С. 1452 - 1471.e10
Опубликована: Окт. 12, 2023
Язык: Английский
Current Oncology, Год журнала: 2023, Номер 30(2), С. 1954 - 1976
Опубликована: Фев. 6, 2023
Gene editing, especially with clustered regularly interspaced short palindromic repeats associated protein 9 (CRISPR-Cas9), has advanced gene function science. editing’s rapid advancement increased its medical/clinical value. Due to great specificity and efficiency, CRISPR/Cas9 can accurately swiftly screen the whole genome. This simplifies disease-specific therapy. To study tumor origins, development, metastasis, change genomes. In recent years, treatment research increasingly employed this method. treat cancer by removing genes or correcting mutations. Numerous preliminary studies have been conducted in relevant fields. may gene-level tumors. CRISPR/Cas9-based personalized targeted medicines shape treatment. review examines for therapy research, which will be helpful providing references future on pathogenesis of malignancy
Язык: Английский
Процитировано
41Pharmaceutics, Год журнала: 2022, Номер 14(6), С. 1252 - 1252
Опубликована: Июнь 13, 2022
Parkinson’s disease (PD) and other chronic debilitating neurodegenerative diseases (NDs) impose a substantial medical, emotional, financial burden on individuals society. The origin of PD is unknown due to complex combination hereditary environmental risk factors. However, over the last several decades, significant amount available data from clinical experimental studies has implicated neuroinflammation, oxidative stress, dysregulated protein degradation, mitochondrial dysfunction as primary causes neurodegeneration. new gene-editing techniques hold great promise for research therapy NDs, such PD, which there are currently no effective disease-modifying treatments. As result, gene may offer treatment options, transforming our ability treat this disease. We present detailed overview novel delivery vehicles, essential their successful implementation in both cutting-edge prospective therapeutics. Moreover, we review most recent advancements CRISPR-based applications therapies better understanding treating PD. explore benefits drawbacks using them range brain, emphasizing some fascinating possibilities.
Язык: Английский
Процитировано
40International Journal of Molecular Sciences, Год журнала: 2023, Номер 24(2), С. 1563 - 1563
Опубликована: Янв. 13, 2023
Human immunodeficiency virus (HIV) infections and HIV-induced acquired syndrome (AIDS) continue to represent a global health burden. There is currently no effective vaccine, nor any cure, for HIV infections; existing antiretroviral therapy can suppress viral replication, but only as long antiviral drugs are taken. infects cells of the host immune system, it establish long-lived reservoir, which be targeted edited through gene therapy. Gene editing platforms based on clustered regularly interspaced palindromic repeat-Cas system (CRISPR-Cas) have been recognized promising tools in development therapies infections. In this review, we evaluate current landscape CRISPR-Cas-based against HIV, with an emphasis infection biology well activity restriction factors. We discuss potential combined CRISPR-Cas approach that targets genes activate factors inhibit replication simultaneously. Lastly, focus challenges solutions approaches achieving cure.
Язык: Английский
Процитировано
33Nature Communications, Год журнала: 2023, Номер 14(1)
Опубликована: Фев. 17, 2023
CRISPR-mediated transcriptional activation (CRISPRa) is a powerful technology for inducing gene expression from endogenous loci with exciting applications in high throughput gain-of-function genomic screens and the engineering of cell-based models. However, current strategies generating potent, stable, CRISPRa-competent cell lines present limitations broad utility this approach. Here, we provide high-efficiency, self-selecting CRISPRa enrichment strategy, which combined piggyBac transposon enables rapid production CRISPRa-ready populations compatible variety downstream assays. We complement an optimized guide RNA scaffold that significantly enhances functionality. Finally, describe synthetic tool set transient, population-wide when used system. Taken together, versatile platform greatly potential across wide cellular contexts.
Язык: Английский
Процитировано
30Cell stem cell, Год журнала: 2023, Номер 30(11), С. 1452 - 1471.e10
Опубликована: Окт. 12, 2023
Язык: Английский
Процитировано
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