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A Visual Guide to Gene Delivery

Codon

Some diseases are caused by mutations in large genes that exceed the packaging limits of existing vectors like adeno-associated virus (AAV). AAV is a small virus that lacks a lipid membrane and carries a single-stranded piece of DNA stretching 4.7 Vectors also differ in the duration of their expression.

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A Visual Guide to Genome Editors

Codon

The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. 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. Visit our website to get a copy. Join Asimov Press.

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Levers for Biological Progress

Codon

Scientists are already building a model that can, for example, look at which RNA molecules are expressed in a cell at t=0 and predict how those molecules will change at t=1. SeqFISH, developed by Long Cai’s group at Caltech, maps the spatial positions of thousands of RNA, DNA, or protein molecules within single cells.

DNA
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AZVUDINE

New Drug Approvals

It was approved in China in 2021 as a first-in-class treatment for human immunodeficiency virus (HIV). It acts as an RNA-dependent RNA polymerase (RdRp) inhibitor, the same mechanism as the previously approved molnupiravir and remdesivir. Detailed numbers are provided by Genuine in the slides and the medication package insert.

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