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2020

CRISPR-Cas9 Genome Editing

Emmanuelle Charpentier & Jennifer A. Doudna

About This Prize

The 2020 Nobel Prize in Chemistry was awarded to Emmanuelle Charpentier and Jennifer A. Doudna “for the development of a method for genome editing.” They discovered that the bacterial immune system CRISPR-Cas9 could be reprogrammed with a synthetic guide RNA to cut DNA at any desired location, creating a revolutionary gene editing tool. CRISPR-Cas9 has transformed biology, enabling precise genome engineering in organisms from bacteria to humans, with applications in medicine, agriculture, and biotechnology.

Emmanuelle Charpentier

“For the Development of a Method for Genome Editing”

Jennifer A. Doudna

“The Chemistry of CRISPR: Editing the Code of Life”

Key Concepts

  • CRISPR-Cas9: Clustered Regularly Interspaced Short Palindromic Repeats — a bacterial adaptive immune system repurposed for genome editing
  • Guide RNA: A programmable ~20-nucleotide RNA sequence that directs Cas9 to complementary DNA targets
  • Double-Strand Break: Cas9 creates a precise DSB, enabling gene knockout (NHEJ) or precise editing (HDR)
  • Therapeutic Applications: Sickle cell disease, beta-thalassemia (Casgevy), cancer immunotherapy, and genetic disease correction