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What CRISPR treatments are FDA approved?Which companies are developing gene-editing treatments for muscular dystrophy?Explain base editing to a 12-year-old.What is the difference between CRISPR and gene therapy?Which gene-editing trials are in Phase III?Which companies are working on in vivo gene editing?What diseases are being targeted by prime editing?How is Casgevy made?原理与局限
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Technology
CRISPR-Cas9
A protein that can be programmed with a short RNA guide to find one specific sequence in a genome…
Treatment
Casgevy (exagamglogene autotemcel)
The first CRISPR-based medicine approved anywhere in the world — a one-time therapy made from a…
Technology
Base Editing
Chemically converts one DNA letter into another at a chosen position, without cutting both strands…
Treatment
Lyfgenia (lovotibeglogene autotemcel)
Approved on the same day as Casgevy for sickle cell disease — but a gene-addition therapy, not gene…
Technology
Prime Editing
Writes a new stretch of DNA sequence directly into a chosen site, using a guide that carries the…
Treatment
Zynteglo (betibeglogene autotemcel)
An approved gene-addition therapy for transfusion-dependent beta thalassemia — the non-editing…
Treatment
Nexiguran ziclumeran (nex-z)
The first CRISPR therapy ever administered into a human bloodstream — a single infusion intended to…
Technology
CRISPR-Cas12
A family of CRISPR nucleases that cut DNA leaving staggered ends, use a different sequence…