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In vivo gene editing achieves another breakthrough, first children's clinical results released

 Last Update: 2021-11-16
 Source: Internet
 Author: User

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Today, LogicBio Therapeutics announced the preliminary results of a Phase 1/2 clinical trial of its in vivo editing therapy LB-001 for the treatment of children with methylmalonic acidemia (MMA)

The press release states that this is the first clinical trial that has shown successful gene editing in children

In vivo gene editing achieved a major breakthrough this year.

The design of LB-001 uses an AAV vector to precisely insert a transgene expressing methylmalonyl-CoA mutase (MMUT) into the albumin gene locus of liver cells

The AAV vector also carries a transgene expressing albumin-2A.

▲The mechanism of action of LB-001 (picture source: LogicBio Therapeutics official website)

Reference materials:

[1] LogicBio Therapeutics Announces Early Clinical Trial Results Demonstrating First-Ever In Vivo Genome Editing in Children.

(The original text has been deleted)

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