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

Broad Institute

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. They also delivered prime editors to the mouse brain, and did not detect any off-target editing.

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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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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. Online June 27, 2024.

Disease 142
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Epigenetic editing: the next generation of genetic medicine

Drug Target Review

The molecular machinery of the epigenetic system can selectively package specific regions of DNA away, making them inaccessible and less active. This platform opens the door to treating complex multigenic diseases, effective tissue engineering, and fine-tuning immune cell differentiation. Molecular Therapy. 2021;29(9):2640–59.

DNA 105
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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. Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs.

DNA 140
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Spectrum Solutions Secures Emergency Use Authorization (EUA) for its SDNA-1000 Saliva Collection Device, Expanding Access and COVID-19 Testing Options

The Pharma Data

Spectrum’s self-contained saliva collection kit provides critical sample consistency while suspending and neutralizing viral RNA transcripts completely inactivating the live virus. Qualified commercial RNA extraction chemistries include Perkin Elmer, Thermo Fisher, Roche, and Qiagen.

RNA 52
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Codon Digest: Discovering Antibiotics with Deep Learning

Codon

Codon Digest is my weekly roundup of research, news, and industry highlights about engineered biology. An engineered version of this protein can convert DNA bases with efficiencies up to 92%. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome.

DNA 52