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

Broad Institute

The studies are part of a package of 10 papers in Nature that take distinct, yet complementary, approaches to mapping the mouse nervous system at the single-cell level. The package also includes the spatial, single-cell atlas of the mouse brain and spinal cord that was led by Wang and Liu and was first published online in September in Nature.

DNA 140
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Scaling Phage Therapy

Codon

doi: 10.2210/rcsb_pdb/goodsell-gallery-048 The Virus that Cures It’s been over 25 years since the science magazine Discover first ran an extraordinary article about how a long-forgotten medical treatment, used in the former Soviet country of Georgia, could save us from the growing threat of untreatable, drug-resistant infections.

Therapies 118
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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

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.

Disease 142
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DNA Barcodes Could Streamline Search for New Drugs to Combat Cancer

NIH Director's Blog: Drug Discovery

Instead of the black, printed stripes of the Universal Product Codes (UPCs) that we see on everything from package deliveries to clothing tags, they used short, unique snippets of DNA to label cells. DNA barcoding has already empowered single-cell analysis, including for nerve cells in the brain.

DNA 52
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Viral Vectors 101: An Introduction to AAV

addgene Blog

Adeno-associated virus (AAV) is a single stranded, Parvoviridae DNA virus, packaged in a non-enveloped icosahedral capsid, that can be used to express genes of interest in cell and animal models.

Virus 53
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Analytical Testing Considerations for Gene Therapy Products

The Premier Consulting Blog

The analytical package, consisting of release, stability, and characterization tests, includes data generated throughout the product development and manufacturing process. For gene therapy vectors, this testing encompasses production cell lines, master and working cell banks (MCB, WCB), and virus banks.