Nobel Prize in Chemistry 2020 for CRISPR-Cas9 Genetic Scissors

On 7 October 2020, the Royal Swedish Academy of Sciences awarded the Nobel Prize in Chemistry 2020 to Emmanuelle Charpentier and Jennifer A. Doudna for the development of a method for genome editing.  Currently Emmanuelle Charpentier works at the Max Planck Unit for the Science of Pathogens in Berlin, Germany, and Jennifer A. Doudna at the University of California, Berkeley, USA. Emanuelle Charpentier [1] and Jennifer A. Doudna [2] Charpentier and team published their initial discovery on bacterial genome...

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Finding the elusive aquaporin modulators

aqua drop
Aquaporins are channel proteins that facilitate the transport of water across cells. The discovery of aquaporins in 1992, for which researcher Peter Agre received the Nobel Prize in Chemistry, opened the door to a new therapeutic approach for treating many health conditions. On one particular start-up’s journey in finding applicable aquaporin modulators, the student has become the master. Water is one of the substances that are essential for life on Earth and for the survival of all plants and animals. Although...

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What are genome editing and CRISPR-Cas9?

Genome editing (also called gene editing) is a set of technologies that enable scientists to change the genetic code in the living cells of an organism. They make it possible to make specific changes to a particular location in the genome in a controlled way. One of the approaches is known as CRISPR-Cas9. The “Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated protein 9”, or CRISPR-Cas9 for short, was discovered by a team of CRISPR pioneers led by biochemists Emmanuelle Charpentier...

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First in vivo CRISPR medicine administered to patients in clinical trial

On 4 March 2020, the collaborating companies Allergan plc and Editas Medicine, Inc. announced the dosing of the first patient with the CRISPR-based medicine AGN-151587 (EDIT-101) in a phase 1/2 clinical trial (BRILLIANCE trial).[1]   Treatment for Leber Congenital Amaurosis 10 AGN-151587 (EDIT-101) is an experimental CRISPR-based medicine currently under development. The medicine is delivered via sub-retinal injection under development for the treatment of Leber congenital amaurosis...

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