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NEI Research News

Thanks to the work of NEI scientists and grantees, we’re constantly learning new information about the causes and treatment of vision disorders. Get the latest updates about their work — along with other news about NEI.

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New gene-editing technique holds potential for treating childhood blindness

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.
Rhodopsin-bound nanobodies

UC Irvine researchers discover a nanobody which may lead to treatment for retinitis pigmentosa

University of California Irvine (UCI) researchers believe they have discovered a special antibody that may lead to a treatment for an inherited eye disease called retinitis pigmentosa.
Eye with coloboma

Zebrafish model helps explain eye development

Developed by NIH scientists, the model also sheds light on developmental eye disorders such as coloboma.
Image of retina

High-tech imaging reveals details about rare eye disorder

Using a new imaging technique, researchers from the National Eye Institute have determined that retinal lesions from vitelliform macular dystrophy (VMD) vary by gene mutation.
Multicolored image of photoreceptors and their long connecting processes, called axons, within a stem cell-derived retinal organoid

U. Wisconsin eye research uncovers how stem cell photoreceptors reach their targets

A new study reveals how photoreceptors grown from stem cells might extend biological wires, known as axons, to contact existing neurons.
Comparison of two retinal images.

Gene therapy for rare eye disease safe but lacks efficacy in early trial

A 28-patient phase 1 gene therapy clinical trial for the degenerative retinal disease Leber hereditary optic neuropathy (LHON) found no significant safety concerns; however, treatment failed to improve or slow vision loss, with even the highest dose.
Group of cells tinted purple in the middle and pink at the edges representing a human induced stem cell colony from a patient with albinism.

NIH researchers develop first stem cell model of albinism to study related eye conditions

Researchers at the National Eye Institute (NEI) have developed the first patient-derived stem cell model for studying eye conditions related to oculocutaneous albinism (OCA).
Rod outer segment showing connector

UC Irvine researchers reveal molecular mechanisms of eye disease using cryo-electron tomography

Understanding the key structural determinants of a highly specialized membrane in the eye could lead to new treatments.
Mother and her daughter smile for the camera

Genomic Study Revealing Among Diverse Populations with Inherited Retinal Disease

International effort identified new and different causative gene variants for a group of diseases that can lead to severe vision loss or blindness.

Gene therapy shows promise in treating rare eye disease in mice

A gene therapy protects eye cells in mice with a rare disorder that causes vision loss, especially when used in combination with other gene therapies, shows a study published in eLife.

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