CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich's Ataxia. [PDF]
Friedreich's ataxia (FRDA) is an autosomal recessive neurodegenerative disorder caused by expansion of GAA repeats in intron 1 of the frataxin (FXN) gene, leading to significant decreased expression of frataxin, a mitochondrial iron-binding protein.
Cherqui, Stephanie +7 more
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Characterisation of dairy strains of Geobacillus stearothermophilus and a genomics insight into its growth and survival during dairy manufacture : a thesis presented in partial fulfilment of the requirements for the degree of Doctor of Philosophy in Microbiology at Massey University, Palmerston North, New Zealand [PDF]
The thermophilic bacilli, such as G. stearothermophilus, are an important group of contaminants in the dairy industry. Although these bacilli are generally not pathogenic, their presence in dairy products is an indicator of poor hygiene and high numbers ...
Burgess, Sara
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Delivering CRISPR: a review of the challenges and approaches
Gene therapy has long held promise to correct a variety of human diseases and defects. Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the mechanism of the CRISPR-based prokaryotic adaptive immune system (CRISPR ...
Christopher A. Lino +3 more
doaj +1 more source
Massively parallel profiling and predictive modeling of the outcomes of CRISPR/Cas9-mediated double-strand break repair. [PDF]
Non-homologous end-joining (NHEJ) plays an important role in double-strand break (DSB) repair of DNA. Recent studies have shown that the error patterns of NHEJ are strongly biased by sequence context, but these studies were based on relatively few ...
Agarwal, Vikram +7 more
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Rapid one-tube RPA-coupled CRISPR/Cas12a-based RID-MyC assay for the diagnosis of fungal keratitis
Purpose: This study introduces and evaluates the single-tube rapid identification of mycoses using CRISPR (ST-RID-MyC) assay. This novel diagnostic tool combines recombinase polymerase amplification (RPA) with CRISPR/Cas12a for the rapid and precise ...
Hanith R Deivarajan +9 more
doaj +1 more source
Mapping genetic interactions in cancer: a road to rational combination therapies. [PDF]
The discovery of synthetic lethal interactions between poly (ADP-ribose) polymerase (PARP) inhibitors and BRCA genes, which are involved in homologous recombination, led to the approval of PARP inhibition as a monotherapy for patients with BRCA1/2 ...
Krogan, Nevan J, Tutuncuoglu, Beril
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Evaluation of behavior in transgenic mouse models to understand human congenital pain conditions [PDF]
BACKGROUND: Containing a brain for signal processing and decision making, and a peripheral component for sensation and response, the nervous system provides higher organisms a powerful method of interacting with their environment.
Bullock, Daniel
core
324 Allogeneic CAR T cells targeting Liv-1 for breast cancer
Minh Pham +9 more
doaj +1 more source
Editing of the MALAT1 Gene in MDA-MB-361 Breast Cancer Cell Line using the Novel CRISPR Method
Introduction: Long non-coding RNAs play an important role in regulating gene expression, RNA processing, histone modification, and rearrangement of chromatin genes. These molecules can also be involved in many biological processes, such as organogenesis,
Soraya Ahmadi-Baloutaki +3 more
doaj

