@usher1f.bsky.social
NPR’s story shows just how deeply the funding decisions affect real lives—and the progress of life-saving science. Craig LeMoult of WGBH in Boston profiles Jessica Chaikof's hope for a cure for Usher 1F and her concern that the federal funding cuts to Harvard threaten Usher 1F research.
She hoped key research could help save her eyesight. Then the Trump funding cuts came
Jessica Chaikof says research into gene therapies could someday save her eyesight. But she worries cuts to federal research funding could mean that therapy won't be ready in time.
www.npr.org
June 25, 2025 at 12:50 AM
NPR’s story shows just how deeply the funding decisions affect real lives—and the progress of life-saving science. Craig LeMoult of WGBH in Boston profiles Jessica Chaikof's hope for a cure for Usher 1F and her concern that the federal funding cuts to Harvard threaten Usher 1F research.
A previously unreported population of human stem-like cells capable of regenerating retinal tissue and potentially promoting vision recovery has been identified.
Retinal cells discovered that could repair vision loss | PET
Stem-like cells have been discovered that might enable retinal repair.
www.progress.org.uk
April 10, 2025 at 2:04 AM
A previously unreported population of human stem-like cells capable of regenerating retinal tissue and potentially promoting vision recovery has been identified.
The research team successfully induced retinal regeneration and vision recovery in a disease-model mouse by administering a compound that blocks the PROX1 (prospero homeobox 1) protein, which suppresses retinal regeneration. Furthermore, the effect lasted for more than six months.
Retinal therapy may restore lost vision
Researchers have successfully developed a novel drug to restore vision. The treatment method restores vision through retinal nerve regeneration.
www.sciencedaily.com
April 3, 2025 at 2:23 PM
The research team successfully induced retinal regeneration and vision recovery in a disease-model mouse by administering a compound that blocks the PROX1 (prospero homeobox 1) protein, which suppresses retinal regeneration. Furthermore, the effect lasted for more than six months.
Exercise can help prevent photoreceptor cell death in retinitis pigmentosa.
Frontiers | Voluntary running partially prevents photoreceptor cell death in retinitis pigmentosa
Retinitis pigmentosa (RP) is a degenerative eye disease characterized by progressive photoreceptor loss. This study evaluates the neuroprotective effects of ...
www.frontiersin.org
April 2, 2025 at 2:33 AM
Exercise can help prevent photoreceptor cell death in retinitis pigmentosa.
Researchers at the NIH have developed eye drops that extend vision in animal models of retinitis pigmentosa.
NIH researchers develop eye drops that slow vision loss in animals
Treatment shows potential to slow the progression of human degenerative eye diseases, including retinitis pigmentosa.
www.nih.gov
March 22, 2025 at 2:18 AM
Researchers at the NIH have developed eye drops that extend vision in animal models of retinitis pigmentosa.
Please contact your senators and representative asking them to stop the Trump administration from blocking medical research. NIH funding is at a standstill, which threatens Usher 1F research and all medical research.
Revealed: NIH research grants still frozen despite lawsuits challenging Trump order
The Trump administration is exploiting a loophole to keep a funding freeze in place, leaving researchers in limbo.
www.nature.com
February 26, 2025 at 2:39 AM
Please contact your senators and representative asking them to stop the Trump administration from blocking medical research. NIH funding is at a standstill, which threatens Usher 1F research and all medical research.
"Regeneron yesterday reported good results with their gene therapy program for children with mutations in otoferlin. 10/11 children had hearing improvement. This is life altering for these children and validation to what we are trying to do with Usher 1F."
Usher 1F board member Frank Gentile, PhD
Usher 1F board member Frank Gentile, PhD
Regeneron gene therapy improves hearing in 10 children with rare form of hearing loss
Regeneron’s investigational gene therapy has been tied to | Regeneron’s investigational gene therapy has been tied to notable hearing improvements in 10 of 11 children who were treated for a rare gene...
www.fiercebiotech.com
February 25, 2025 at 11:53 PM
"Regeneron yesterday reported good results with their gene therapy program for children with mutations in otoferlin. 10/11 children had hearing improvement. This is life altering for these children and validation to what we are trying to do with Usher 1F."
Usher 1F board member Frank Gentile, PhD
Usher 1F board member Frank Gentile, PhD
"It’s a crucial moment not just for the three companies and the drugs, but also a chance to remove what’s long been seen as a major roadblock to developing treatments for ultra-rare disease populations."
Drugmakers seize on biomarkers to test the FDA's rare disease shift
For the first time, the FDA will consider whether to approve a trio of drugs for tiny patient populations based on trials that are using biological signals as outcomes, rather than a placebo compariso...
endpts.com
February 3, 2025 at 4:46 PM
"It’s a crucial moment not just for the three companies and the drugs, but also a chance to remove what’s long been seen as a major roadblock to developing treatments for ultra-rare disease populations."
Our 2024 Usher 1F Collaborative Annual Report is available. We had a banner year! Thank you so much to all our supporters. www.usher1f.org/file_downloa...
January 23, 2025 at 5:21 PM
Our 2024 Usher 1F Collaborative Annual Report is available. We had a banner year! Thank you so much to all our supporters. www.usher1f.org/file_downloa...
