Gene editing in hemophilia: a “CRISPR” choice?
نویسندگان
چکیده
منابع مشابه
Core Concept: CRISPR gene editing.
Just a few years ago, molecular biologists hoping to alter the genome of their favorite organisms faced an arduous task and likely weeks of genetic tinkering. Today, those scientists can quickly destroy or edit a gene with a new technology called CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9. “It really opens up the genome of virtually every organism that’s been sequenc...
متن کاملCRISPR as a strong gene editing tool
Clustered regularly-interspaced short palindromic repeats (CRISPR) is a new and effective genetic editing tool. CRISPR was initially found in bacteria to protect it from virus invasions. In the first step, specific DNA strands of virus are identified by guide RNA that is composed of crRNA and tracrRNA. Then RNAse III is required for producing crRNA from pre-crRNA. In The second step, a crRNA:tr...
متن کاملThe new genomic editing system (CRISPR)
Over the past decades, progression in genetic element manipulation, and consequently, the treatment of diseases has been remarkable. It is worth noting that these genetic manipulations perform at different levels, including DNA and RNA. The earlier genomic editing techniques, including MN, ZFN , TALEN , performing their functions by creating double-stranded breaks (DSBs), and after breakage, th...
متن کاملUSH2A Gene Editing Using the CRISPR System
Usher syndrome (USH) is a rare autosomal recessive disease and the most common inherited form of combined visual and hearing impairment. Up to 13 genes are associated with this disorder, with USH2A being the most prevalent, due partially to the recurrence rate of the c.2299delG mutation. Excluding hearing aids or cochlear implants for hearing impairment, there are no medical solutions available...
متن کاملCRISPR - Cas 9 : Engineering a Revolution in Gene Editing
Cover image provided courtesy of Horizon Discovery. This image has been digitally altered from the original version for publication purposes.
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ژورنال
عنوان ژورنال: Blood
سال: 2019
ISSN: 0006-4971,1528-0020
DOI: 10.1182/blood.2019001180