DNA Engineering Packaging 
Broad Institute
JANUARY 14, 2025
The treatment, which uses base editing to make a single-letter change in DNA, reduced levels of the disease-causing prion protein in the brain by as much as 60 percent. In 2018, Liu, who works on the same floor as Minikel and Vallabh at Broad, approached them and proposed a collaboration.
Broad Institute
JANUARY 8, 2024
A team of researchers at the Broad Institute of MIT and Harvard has engineered virus-like particles to deliver prime editors to cells in mice at a high enough efficiency to rescue a genetic disorder. Prime editing, described in 2019 by Liu’s group, can make longer and more diverse types of DNA changes than other types of editing.
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Broad Institute
JULY 10, 2024
The new method, published today in Nature Biomedical Engineering , precisely and durably corrects the mutation in human lung cells, restoring cell function to levels similar to that of Trikafta. Next, researchers will need to develop ways to package and deliver the prime editing machinery to the airways in mice and ultimately humans.
Broad Institute
JUNE 27, 2024
Unlike gene editing, this “epigenetic” editing does not modify the underlying DNA sequence, but it should switch the gene off permanently, which means that this could be a one-time treatment. They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration. Online June 27, 2024.
Drug Target Review
JULY 7, 2023
The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?
Broad Institute
DECEMBER 13, 2023
The other study was led by senior authors Xiao Wang , a Broad core institute member, a Merkin Institute Fellow, and an assistant professor of chemistry at MIT, and Jia Liu, an assistant professor of engineering at Harvard University. The team next applied an approach developed in the Chen and Macosko labs known as Slide-seq.
Drug Target Review
JUNE 9, 2023
As part of the TCR discovery process and preclinical data package, Adaptive performs rigorous functional characterisation and safety assessments of potent, therapeutic grade antigen-specific TCRs. Genentech is responsible for engineering adaptive-discovered TCRs into T cells and manufacturing the T cell therapy product candidate.
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