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Genome editing 

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas genome editing tools have built on experience with previous tools such as Zinc Finger Nucleases (ZFNs) and TALE nucleases (TALENS).

 

Nuclease-free versions that chemically change single nucleotides by deamination to ‘base-edit’ genes without the need for DNA break have reached clinical stage testing. Prime-editing is also under investigation for more complex editing

 

Our goal is to identify and advance the most promising technologies into clinical phase testing to address unmet medical needs

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