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Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Broad Institute

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. By Sarah C.P. However, delivering the complex gene editing machinery to cells in living animals has been challenging.

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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

Building on previous work by the vector-engineering lab of Ben Deverman at the Broad, the team developed a pair of adeno-associated viruses (AAVs) to package and deliver the base-editing machinery to brain cells. But the researchers needed to deliver the base editors to the brain.

Disease 144
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New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor. This engineered capsid opens up a world of possibilities."

Therapies 137
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Researchers reprogram gene therapy viral vectors to bind specific protein targets

Broad Institute

By Allessandra DiCorato July 19, 2023 Credit: Kevin Middleton, Broad Communications A three-dimensional model of adeno-associated viruses (AAVs), which scientists have engineered to package and deliver gene therapies to cells in the body. Qin Huang, a senior research scientist in Deverman’s group, is the first author on the study.

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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

To deliver CHARM throughout the brain, the team collaborated with scientists in the lab of Ben Deverman , senior director of vector engineering at the Broad and a co-author of the study. They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration.

Disease 142
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Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy

Broad Institute

Existing methods of engineering one of the most commonly used gene-delivery vehicles, adeno-associated viruses (AAV), are often slow and inefficient. Now, researchers at the Broad Institute of MIT and Harvard have developed a machine-learning approach that promises to speed up AAV engineering for gene therapy. Nature Communications.

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Scientists map the locations of hundreds to thousands of cell types across a mammalian brain

Broad Institute

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. Our atlas represents the largest spatial single-cell dataset our lab has analyzed to date,” said Wang.

DNA 140