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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.

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A Visual Guide to Genome Editors

Codon

If the same virus invades the cell a second time, the gRNA’s spacer sequence will bind to the matching viral DNA sequence, then be cut by the Cas protein. pyogenes protein — whose compactness makes them far easier to package into viral vectors and deliver into the human body.

DNA
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Levers for Biological Progress

Codon

Making a computer-designed protein in the laboratory requires, first, that scientists synthesize a string of DNA encoding the protein, insert that DNA into living cells, and then isolate the proteins churned out by the engineered cells. The world’s supply of insulin is now made by engineered microbes.

DNA
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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."

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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.