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<a href="/about/news-and-events/news?topic=All#block-nei-content" class="c-filter__pill">Gene Therapy</a><a href="/about/news-and-events/news#block-nei-content" class="c-filter__pill--clear">Clear all filters</a>
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<div class="field-content"><div class="c-teaser">
<div class="c-teaser__image">
<img loading="lazy" src="/sites/default/files/styles/thumbnail/public/2024-12/optic-nerves-998x665.jpg?itok=xeekyoXZ" width="200" height="200" alt="optic nerves" />
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<a class="c-teaser__link" href="/about/news-and-events/news/nerve-regrowth-sight">
Nerve regrowth in sight
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</h2>
<p class="c-teaser__date">
<time datetime="2024-12-05T14:50:42-05:00">December 5, 2024</time>
</p>
<div class="c-teaser__summary">
UConn scientists led by Ephraim Trakhtenberg regrew optic nerves in mice through eye injections of fibronectin peptides (pieces of the larger protein).
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<img loading="lazy" src="/sites/default/files/styles/thumbnail/public/2024-11/layers%20of%20retina.jpeg?itok=l95gwxMc" width="200" height="200" alt="" />
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<h2 class="c-teaser__header">
<a class="c-teaser__link" href="/about/news-and-events/news/crispr-gene-editing-promising-blinding-disease-retinitis-pigmentosa">
CRISPR gene editing promising for blinding disease retinitis pigmentosa
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</h2>
<p class="c-teaser__date">
<time datetime="2024-11-27T09:25:51-05:00">November 27, 2024</time>
</p>
<div class="c-teaser__summary">
National Eye Institute-funded scientists using gene editing corrected a mutation in a mouse model of retinitis pigmentosa, an inherited blinding eye disorder. The gene editing strategy restored production of rhodopsin, and retinal function and structure
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<img loading="lazy" src="/sites/default/files/styles/thumbnail/public/news/LCA_disorder.jpg?itok=pO9qcOct" width="200" height="200" alt="LCA is an inherited disorder that causes vision loss in childhood. It primarily affects the functioning of the retina, the light-sensitive tissue at the back of the eye, as shown here. Photo credit: National Eye Institute" />
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<h2 class="c-teaser__header">
<a class="c-teaser__link" href="/about/news-and-events/news/participants-pioneering-crispr-gene-editing-trial-see-vision-improve">
Participants of pioneering CRISPR gene editing trial see vision improve
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</h2>
<p class="c-teaser__date">
<time datetime="2024-05-06T11:09:39-04:00">May 6, 2024</time>
</p>
<div class="c-teaser__summary">
About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness.
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<img loading="lazy" src="/sites/default/files/styles/thumbnail/public/2024-03/subretinal-injection_creditOSU_thumb.png?itok=2C6o7P6O" width="200" height="200" alt="" />
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<h2 class="c-teaser__header">
<a class="c-teaser__link" href="/about/news-and-events/news/researchers-closing-genetic-treatments-hereditary-lung-disease-vision-loss">
Researchers closing in on genetic treatments for hereditary lung disease, vision loss
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</h2>
<p class="c-teaser__date">
<time datetime="2024-03-04T12:39:56-05:00">March 4, 2024</time>
</p>
<div class="c-teaser__summary">
Researchers who work with tiny drug carriers known as lipid nanoparticles have developed a new type of material capable of reaching the lungs and the eyes, an important step toward genetic therapy for hereditary conditions like inherited vision loss.
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<img loading="lazy" src="/sites/default/files/styles/thumbnail/public/2024-01/canine-blindness_creditTaskinUPenn.jpeg?itok=9AhLpkQw" width="200" height="200" alt="fMRI of dog brain showing bright activation in the visual cortex." />
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<h2 class="c-teaser__header">
<a class="c-teaser__link" href="/about/news-and-events/news/using-fmri-new-vision-study-finds-promising-model-restoring-cone-function">
Using fMRI, new vision study finds promising model for restoring cone function
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</h2>
<p class="c-teaser__date">
<time datetime="2024-01-26T09:50:55-05:00">January 26, 2024</time>
</p>
<div class="c-teaser__summary">
Researchers have found that in dogs, fMRI can detect brain responses to daylight vision for black and white information as well as color information, and identify the area of the visual cortex that responds to stimulation of a cone-rich retinal region.
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<img loading="lazy" src="/sites/default/files/styles/thumbnail/public/2019-02/normalvspigmentosas.jpg?itok=8Vj-X-GU" width="200" height="200" alt="The image on the left is labeled &quot;Normal retina,&quot; and shows green rods and red cones. The image on the right is labeled &quot;Retina affected by retinitis pigmentosa,&quot; and shows green rods that have broken down." />
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<h2 class="c-teaser__header">
<a class="c-teaser__link" href="/about/news-and-events/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice">
Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice
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</h2>
<p class="c-teaser__date">
<time datetime="2024-01-08T11:11:25-05:00">January 8, 2024</time>
</p>
<div class="c-teaser__summary">
By adapting virus-like particles to carry the machinery for a type of gene editing called prime editing, scientists have corrected disease-causing mutations in animals and increased editing efficiency.
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<img loading="lazy" src="/sites/default/files/styles/thumbnail/public/2023-10/Nanoparticle.jpg?itok=on1V4UTs" width="200" height="200" alt="" />
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<h2 class="c-teaser__header">
<a class="c-teaser__link" href="/about/news-and-events/news/new-gene-editing-technique-holds-potential-treating-childhood-blindness">
New gene-editing technique holds potential for treating childhood blindness
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</h2>
<p class="c-teaser__date">
<time datetime="2023-10-05T09:20:24-04:00">October 5, 2023</time>
</p>
<div class="c-teaser__summary">
Using a new experimental technique to fix faulty eye cells, a team led by University of Wisconsin researchers was able to repair a gene mutation that causes one form of childhood blindness.
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<img loading="lazy" src="/sites/default/files/styles/thumbnail/public/2023-04/Dog.jpg?itok=q3QqtyCA" width="200" height="200" alt="" />
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<h2 class="c-teaser__header">
<a class="c-teaser__link" href="/about/news-and-events/news/precision-eye-therapy-dogs-ready-human-clinical-development">
Precision eye therapy for dogs ready for human clinical development
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</h2>
<p class="c-teaser__date">
<time datetime="2023-04-28T09:37:23-04:00">April 28, 2023</time>
</p>
<div class="c-teaser__summary">
A successful gene therapy trialed at Michigan State University in dogs with an inherited eye disease is ready to be developed for clinical use in human patients with a rare condition called retinitis pigmentosa.
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<img loading="lazy" src="/sites/default/files/styles/thumbnail/public/2023-04/child%20eye%20exam.jpg?itok=6SiRSy4A" width="200" height="200" alt="" />
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<h2 class="c-teaser__header">
<a class="c-teaser__link" href="/about/news-and-events/news/university-houston-researcher-builds-new-model-examine-usher-syndrome">
University of Houston researcher builds new model to examine Usher syndrome
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</h2>
<p class="c-teaser__date">
<time datetime="2023-04-18T15:04:58-04:00">April 18, 2023</time>
</p>
<div class="c-teaser__summary">
Syndrome is a leading cause of combined deafness-blindness
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<div class="field-content"><div class="c-teaser">
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<img loading="lazy" src="/sites/default/files/styles/thumbnail/public/2023-01/Lipid-nanoparticle-based-gene-therapy_creditOHSU.jpg?itok=wpJ7U5Zd" width="200" height="200" alt="3D illustration of nanoparticle inside an eye" />
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<div class="c-teaser__content">
<h2 class="c-teaser__header">
<a class="c-teaser__link" href="/about/news-and-events/news/nanotechnology-may-improve-gene-therapy-blindness">
Nanotechnology may improve gene therapy for blindness
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</h2>
<p class="c-teaser__date">
<time datetime="2023-01-11T17:24:24-05:00">January 11, 2023</time>
</p>
<div class="c-teaser__summary">
Using nanotechnology that enabled mRNA-based COVID-19 vaccines, a new approach to gene therapy may improve how physicians treat inherited forms of blindness.
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