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One tool, called Find My Understudied Genes (FMUG), emerged from a study published in March¹, which first explores why interesting, but relatively under-researched, genes are not highlighted in genetic surveys, and then offers FMUG as a remedy.

The second tool is the Unknome database, created by a team led by Matthew Freeman at the University of Oxford, UK, and Sean Munro at the MRC Laboratory of Molecular Biology, Cambridge, UK, that was described² in 2023.

“We are in the lucky position to know what we don’t know,” says Thomas Stoeger, a biologist at Northwestern University in Chicago, Illinois, and co-author of the FMUG study.

New study could improve the ability to make genetic diagnoses and treat neurodevelopmental disorders.

Imagine a technology that could genetically rewire organisms in real-time, silencing critical genes across entire ecosystems with unknown effects.

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Scientists have pinpointed genetic changes that can leave children born with little to no immune defense against infection.

Retinitis pigmentosa and macular degeneration lead to photoreceptor death and loss of visual perception. Despite recent progress, restorative technologies for photoreceptor degeneration remain largely unavailable. Here, we describe a novel optogenetic visual prosthesis (FlexLED) based on a combination of a thin-film retinal display and optogenetic activation of retinal ganglion cells (RGCs). The FlexLED implant is a 30 µm thin, flexible, wireless µLED display with 8,192 pixels, each with an emission area of 66 µm2. The display is affixed to the retinal surface, and the electronics package is mounted under the conjunctiva in the form factor of a conventional glaucoma drainage implant. In a rabbit model of photoreceptor degeneration, optical stimulation of the retina using the FlexLED elicits activity in visual cortex. This technology is readily scalable to hundreds of thousands of pixels, providing a route towards an implantable optogenetic visual prosthesis capable of generating vision by stimulating RGCs at near-cellular resolution.

### Competing Interest Statement.

All authors have a financial interest in Science Corporation.

Although the Human Genome Project announced the completed sequencing of 20,000 human genes more than 20 years ago, scientists are still working to grasp how fully formed beings emerge from basic genetic instructions.

Prime editing, a mightier version of CRISPR/Cas9 technology, has been part of rigorous research and development in recent years. Now, U.S. regulators have greenlit the first-ever clinical trial for this technology.

Massachusetts-based Prime Medicine received the go-ahead from the U.S. Food and Drug Administration (FDA) after preclinical data showed that its candidate was able to correct mutations in chronic granulomatous disease (CGD).

CGD is a rare condition and affects around one in 200,000 people worldwide. It is caused by mutations in any of the six genes that code for the molecule nicotinamide adenine dinucleotide phosphate (NADPH), which is responsible for carrying electrons within cells. White blood cells called phagocytes don’t function properly, and as a result, they fail to protect the body from bacterial and fungal infections.

The ability to genetically modify haematopoietic stem cells would allow the durable treatment of a diverse range of genetic disorders but gene delivery to the bone marrow has not been achieved. Here lipid nanoparticles that target and deliver mRNA to 14 unique cells within the bone marrow are presented.