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Experimental work by several groups revealed the basic mechanisms of CRISPR-Cas immunity. In 2007, the first experimental evidence that CRISPR was an adaptive immune system was published. [6] [12] A CRISPR region in Streptococcus thermophilus acquired spacers from the DNA of an infecting bacteriophage.
CRISPR Therapeutics (CRSP) receives clearance from Health Canada to initiate clinical studies for VCTX210, its investigational therapy for the treatment of type 1 diabetes.
CRISPR interference (CRISPRi) is a genetic perturbation technique that allows for sequence-specific repression of gene expression in prokaryotic and eukaryotic cells. [1] It was first developed by Stanley Qi and colleagues in the laboratories of Wendell Lim , Adam Arkin, Jonathan Weissman , and Jennifer Doudna . [ 2 ]
CRISPR RNA or crRNA is a RNA transcript from the CRISPR locus. [1] CRISPR-Cas (clustered, regularly interspaced short palindromic repeats - CRISPR associated systems) is an adaptive immune system found in bacteria and archaea to protect against mobile genetic elements , like viruses , plasmids , and transposons . [ 2 ]
Immunogenetics or immungenetics is the branch of Medical Immunology and Medical Genetics that explores the relationship between the immune system and genetics.. Autoimmune diseases, such as type 1 diabetes, are complex genetic traits which result from defects in the immune system.
CRISPR-based gene knockout is a powerful tool for understanding the genetic basis of disease and for developing new therapies. It is important to note that CRISPR-based gene knockout, like any genetic engineering technique, has the potential to produce unintended or harmful effects on the organism, so it should be used with caution.
Off-target genome editing refers to nonspecific and unintended genetic modifications that can arise through the use of engineered nuclease technologies such as: clustered, regularly interspaced, short palindromic repeats ()-Cas9, transcription activator-like effector nucleases (), meganucleases, and zinc finger nucleases (ZFN). [1]
With the discovery of various types of immune-related disorders, there is a need for diversification in prevention and treatment. Developments in the field of gene therapy are being studied to be included in the scope of this treatment, but of course more research is needed to increase the positive results and minimize the negative effects of gene therapy applications. [27]