The study marks milestone progress over previous attempts to remodel the human embryos DNA. It work well in embryos created by a standard in-vitro fertilization technique called "germline editing". The research is published last week in the journal Molecular Therapy.
Chinese scientists corrected a mutation that causes Marfan syndrome, an incurable connective tissue disorder that affects about 1 in 5,000 people. A single letter mistake in the gene for FBN1, which codes for the fibrillin protein, can cause ripple effect problems-from loose joints to weak vision to life-threatening heart damage. The team of researchers from Shanghai Tech University and Guangzhou Medical University used an IVF technique to make viable human embryos. Then they injected the embryos with a CRIPR construct known as a base editor, which swaps out a single DNA nucleotide for another-n this case, removing an "A" and replacing it with a "G".
Sequencing revealed that all 18 embryos had been edited, with 16 of the embryos corrected version of the FBN1 gene. In two of the embryos, additional unwanted edits had also taken place.
GenScript synthesizes both unmodified and modified sgRNAs for targeted genome editing. Modified sgRNAs with 2’-O-methyl and phosphorothioate modifications at the first three 5’ and 3’ terminal residues are recommended for better stability and editing efficiency.
