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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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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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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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Cas9 ancestor engineered into a compact genome editing tool

Broad Institute

Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells.

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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
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Delivering gene-based therapies

Broad Institute

Some are focused on viruses called adeno-associated viruses (AAVs), which have been engineered to act only as a one-time delivery vehicle and not cause disease. Broad researchers are further engineering AAVs to efficiently deliver gene-based therapies to tissues that have been difficult to reach, such as the muscle and brain.