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Therapies based on CRISPR–Cas3 gene editing technology delivered by engineered bacteriophages could be used to destroy targeted DNA in pathogens. [194] Cas3 is more destructive than the better known Cas9. [195] [196] Research suggests that CRISPR is an effective way to limit replication of multiple herpesviruses.
Cure Rare Disease is a non-profit biotechnology company based in Boston, Massachusetts that is working to create novel therapeutics using gene therapy, gene editing (CRISPR technology) and antisense oligonucleotides to treat people impacted by rare and ultra-rare genetic neuromuscular conditions.
CRISPR technology is a promising tool not only for genetic disease corrections but also for the prevention of viral and bacterial infections. Utilizing CRISPR–Cas therapies, researchers have targeted viral infections like HSV-1, EBV, HIV-1, HBV, HPV, and HCV, with ongoing clinical trials for an HIV-clearing strategy named EBT-101 ...
[124] [125] One possible solution is to add a functional tumor suppressor gene to the DNA to be integrated. This may be problematic since the longer the DNA is, the harder it is to integrate into cell genomes. [126] CRISPR technology allows researchers to make much more precise genome changes at exact locations. [127]
Victoria Gray was the first patient ever to be treated with the gene-editing tool CRISPR for sickle-cell disease. [1]This marked the initial indication that a cure is attainable for individuals born with sickle-cell disease and another severe blood disorder, beta-thalassemia.
CRISPR gene editing is a revolutionary technology that allows for precise, targeted modifications to the DNA of living organisms. Developed from a natural defense mechanism found in bacteria, CRISPR-Cas9 is the most commonly used system, that allows "cutting" of DNA at specific locations and either delete, modify, or insert genetic material.
Fast-paced developments in the CRISPR-Cas9 gene editing technology has increased both the concerns and relevance of this ethical controversy as it has become more popularly used. [19] [20] The scientific community recommends continued evaluation of risks and benefits of utilizing genetically modified organisms in everyday life. [21]
Genome editing is characterised by making small edits to the genome at a specific location, often following cutting of the target DNA region by a site-specific-nuclease such as CRISPR. [12] Genetic modification usually describes the insertion of a transgene (foreign DNA, i.e. a gene from another species) into a random location within the genome.