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New study highlights lipid nanoparticles and mRNA could treat blindness
The researchers developed nanoparticles able to penetrate the neural retina and deliver mRNA to the photoreceptor cells whose proper function makes vision possible.
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3D map reveals DNA organisation within human retina cells
Scientists shed light on how genetic architecture in human retina cells determine gene expression, tissue-specific function, and disease phenotype in blinding diseases.
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Researchers develop NPHP5 gene therapy for rare ciliopathy
The researchers found that augmenting NPHP5 gene rescues cilia defects in light-sensing cells derived from ciliopathy patients.
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Progress towards a stem cell-based therapy for blindness
Scientists are developing an effective stem cell-based therapy to regenerate photoreceptors cells and restore sight in people with blindness.
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Improving eye treatment delivery with microneedles
Scientists have developed self-plugging microneedles that could improve delivery of drugs into the eyeball.
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Retinal cell map could advance precise cell and gene therapies for retinal diseases
Researchers have identified distinct differences among the cells comprising a tissue in the retina, findings that could help develop precise therapies for retinal diseases.
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Novel DNA-free virus-like particles deliver gene-editing proteins to animal models
Researchers have developed a new drug delivery system using virus-like particles to successfully transport gene-editing proteins in pre-clinical studies.
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CRISPR editing can result in whole chromosome elimination, say scientists
In a new study, scientists identify some of the pitfalls when using CRISPR Cas9 to correct mutations in human embryos, such as the destruction of whole chromosomes.
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Study shows how cone cells in the retina form connections with brain
Researchers have discovered how cone cells in the retina send information to the brain, which could be used in the development of treatments for blindness.
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Novel cell reprogramming cocktail could advance regenerative medicine
Scientists have showed that a three inhibitors (3i) cocktail could reprogramme fibroblasts to a naïve embryonic stem cell-like state and remove disease-associated epigenetic changes.
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Novel molecules to adjust the circadian rhythm
A collaborative team of researchers has discovered two molecules that target the melatonin receptor, responsible for regulating the circadian rhythm. This article delves into their study and its therapeutic relevance for the body clock.
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Membrane protein function behind epilepsy discovered
Researchers have revealed the 3D structure of a membrane protein which plays a role in the development of conditions such as epilepsy and blindness.
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Recessive disorder impairing eyesight has been discovered
Blindness in children has been linked to a recessive genetic disorder caused by the MARK3 gene...
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GABA shown to trigger stem cell production in the retina
Research in zebrafish opens up new ideas for therapies for blinding diseases