2016
DOI: 10.1136/archdischild-2016-310459
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What is CRISPR/Cas9?

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Cited by 112 publications
(79 citation statements)
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References 13 publications
(18 reference statements)
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“…The use of site-speciic DNA endonucleases including Zinc inger nucleases (ZFNs), [161] transcription activator-like efector nucleases (TALENs) [162] and now clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 [163] have equipped researchers with the ability to speciically inactivate genes and target genetic regions for homologous recombination of input DNA. In general, double-stranded breaks introduced by nucleases activates DNA repair mechanisms which generate mutations in the target sequence conferring a loss of expression i.e., gene editing.…”
Section: Host Resistancementioning
confidence: 99%
“…The use of site-speciic DNA endonucleases including Zinc inger nucleases (ZFNs), [161] transcription activator-like efector nucleases (TALENs) [162] and now clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 [163] have equipped researchers with the ability to speciically inactivate genes and target genetic regions for homologous recombination of input DNA. In general, double-stranded breaks introduced by nucleases activates DNA repair mechanisms which generate mutations in the target sequence conferring a loss of expression i.e., gene editing.…”
Section: Host Resistancementioning
confidence: 99%
“…Die HK ist somit eine seltene Erbkrankheit, die allerdings für diese Krankheitskategorie vergleichsweise viele Menschen betrifft. Da immer mehr Deutsche ein hohes Lebensalter erreichen und somit lange genug leben, um eine klinische Manifestation der HK auch bei geringen CAG-Triplett-Wiederholungen (36)(37)(38)(39) im HTT-Gen zu erlauben, ist eine Zunahme der Prävalenz wahrscheinlich [4].…”
Section: Hintergrundunclassified
“…In 2005, various research groups reported that CRISPR was observed in prokaryotes and supported the acquired immunity to viruses (Bolotin et al, 2005;Pourcel et al, The CRISPR-Cas system, originally described as an adaptive immune system in bacteria and archaea, is now designed as RNA-directed endonucleases for genome regulation, enabling rapid, inexpensive and relatively easy correction of errors in the genome. (Ma et al, 2014;Redman et al, 2016). In this technology, programmable nucleases, similar to DNA restriction enzymes, cuts DNA double strands in the region where genome editing is desired.…”
Section: Genome Editing and Crispr-cas9mentioning
confidence: 99%
“…Recently, significant progress has been made in this area. One of the most exciting applications of CRISPR-Cas9 is its use for the treatment of genetic diseases caused by single gene mutations such as Duchenne muscle dystrophy (DMD), cystic fibrosis (CF) and hemoglobinopathies (Dai et al, 2016;Redman et al, 2016). There are several studies related to the in vivo genome editing for the repair of DMD-causing mutations in the dystrophic mouse model published in Science.…”
Section: Genome Editing and Crispr-cas9mentioning
confidence: 99%