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CRISPR started out as a defence system in bacteria, which store snippets of virus DNA and cut up any virus that matches them. In 2012 Jennifer Doudna and Emmanuelle Charpentier showed it could be programmed to cut DNA at almost any chosen spot, which made editing genes far easier.

Nobel Prize / CRISPR research

TechnologyScience

The story behind the card

The name stands for ‘clustered regularly interspaced short palindromic repeats’: short DNA sequences repeated at regular intervals, with the virus snippets stored between them. Japanese researchers first noticed the pattern in 1987, and the Spanish microbiologist Francisco Mojica later worked out that it serves as an immune memory. The editing tool pairs a cutting protein, Cas9, with a short guide made of RNA, DNA's chemical cousin, that leads it to the matching DNA. When the cell repairs the cut, a gene can be switched off, or scientists can slip in a corrected version. Doudna and Charpentier won the 2020 Nobel Prize in Chemistry. In November 2023 the UK became the first country to approve a CRISPR treatment: Casgevy, for the inherited blood disorders sickle cell disease and beta thalassaemia. The method also raises ethical questions: in 2018 the Chinese scientist He Jiankui edited human embryos and was later jailed.

Türkçe oku
CRISPR, bakterilerin virüslere karşı savunma sistemi olarak doğdu: Bakteri, virüs DNA'sından parçalar saklar ve bunlara uyan virüsü tanıyıp keser. 2012'de Jennifer Doudna ile Emmanuelle Charpentier, sistemin DNA'yı hemen her noktadan kesecek şekilde programlanabileceğini gösterdi; böylece gen düzenlemek çok kolaylaştı.

CRISPR, İngilizcede “kümelenmiş, düzenli aralıklı, kısa palindromik tekrarlar” anlamına gelen adın kısaltmasıdır. Ad, bakteri DNA'sında belli aralıklarla yinelenen kısa dizileri anlatır; virüs parçaları bu tekrarların arasında saklanır. Düzeni ilk kez 1987'de Japon araştırmacılar fark etti, İspanyol mikrobiyolog Francisco Mojica da sonradan bunun bir tür bağışıklık belleği olduğunu ortaya koydu. Düzenleme aracında Cas9 adlı kesici bir protein, DNA'nın kimyasal akrabası RNA'dan yapılmış kısa bir kılavuzla çalışır; kılavuz onu DNA'nın eşleşen bölümüne götürür. Hücre kesiği onarırken gen devre dışı kalabilir ya da bilim insanları yerine düzeltilmiş bir kopya yerleştirebilir. Doudna ile Charpentier 2020 Nobel Kimya Ödülü'nü aldı. Kasım 2023'te Birleşik Krallık bir CRISPR tedavisini onaylayan ilk ülke oldu: Casgevy adlı tedavi iki kalıtsal kan hastalığında, orak hücre hastalığında ve Akdeniz anemisi olarak da bilinen beta talasemide kullanılıyor. Yöntem etik soruları da beraberinde getirdi: Çinli bilim insanı He Jiankui 2018'de insan embriyolarının genlerini değiştirdi ve sonradan hapis cezası aldı.

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Background reading: CRISPR
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