Our research mentioned in the media

• January 2022 (re CEP290-LCA)
  • Gilmore Health News: Sepofarsen a Wonder Drug That Reverses Blindness in a Single Shot
• December 2021 (re CEP290-LCA)
  • Scheie Vision: RNA therapy reverses blindness in patient after one dose
    • Cover image
• July 2021 (re Retinal gene therapy)
  • Retina Today: Let’s talk about gene therapy for inherited retinal diseases
    • New Retina Radio Podcast: The Latest on Gene Therapy for Inherited Retinal Diseases
• April 2021 (re GUCY2D-LCA)
  • European Pharmaceutical Review: Novel blindness gene therapy shows promise in first-in-human trial
  • News Medical: Gene therapy for common forms of congenital blindness shows promise to improve vision
  • Health IT Analytics: Precision medicine therapy improves congenital blindness
  • Penn News: Gene therapy shows promise in initial trial for patients with childhood blindness
  • FFB: Encouraging early report for three patients in LCA1-GUCY2D gene therapy clinical trial
• April 2021 (re CEP290-LCA)
  • Drug Discovery & Development: How ProQR aims to use RNA therapy to target genetic forms of blindness
  • Drug Discovery & Development: Investigational drug gives sight to patient with inherited form of blindness 
  • The Ophthalmologist: Expanding tunnel vision
  • ProQR: ProQR announces publication in Nature Medicine for sepofarsen in Leber congenital amaurosis 10
  • Freethink: Penn scientists correct genetic blindness with a single injection into the eye
  • The Daily Pennsylvanian: Penn Medicine researchers successfully test experimental therapy to reverse blindness
  • Ophthalmology Times: In clinical trial, injection improves vision in a form of childhood blindness
  • The Hill: Single shot of this groundbreaking drug reversed patient's blindness
  • Science Times: Gene therapy gives vision to visually impaired patients since childhood, study reveals
  • GEN: Mutation-specific RNA therapy can improve vision with lasting effects
  • UNILAD: Experimental shot restored blind child’s vision for a year
  • Futurism.com: Gene therapy injected into eyeball restores blind patient's vision
  • Clinical Omics: Genetically-caused blindness reversed with single injection of antisense therapeutic
  • Penn News: A single injection reverses blindness in patient with rare genetic disorder
• March 2021 (re NPHP5-LCA)
  • The Ophthalmologist: Reversing LCA?
  • NEI: Turning back the clock on a severe vision disorder
  • Penn News: Turning back the clock on a severe vision disorder
• July 2020 (re RHO-ADRP)
  • FFB: Researchers identify regions in the retina to target therapies for certain RP patients
• May 2020 (re RPGR-XLRP)
  • AGTC: AGTC announces publication of positive preclinical data that provides foundational scientific evidence for its ongoing clinical program in patients with X-linked retinitis pigmentosa (XLRP)
• March 2020
  • NPR: In a 1st, scientists use revolutionary gene-editing tool to edit inside a patient
  • Nature News: CRISPR treatment inserted directly into the body for first time
• September 2019 (re RPE65-LCA)
  • Penn News: Is treatment forever? Success of gene therapy for inherited blindness depends on timing.
  • EurekAlert: Success of gene therapy for a form of inherited blindness depends on timing
  • docwire: Effectiveness of gene therapy for blindness depends on timing
  • OT: Timing key in administering gene therapy
• January 2019 (re CEP290-LCA)
  • The Ophthalmologist: From Darkness
  • MD Magazine: Early gene therapy trials for retina disorder yield promise
• December 2018 (re CEP290-LCA)
  • Penn Medicine News: Injection improves vision in a form of childhood blindness
  • ProQR Therapeutics: ProQR announces publication in Nature Medicine of QR-110 data for Leber’s congenital amaurosis 10
  • GEN: Injectable RNA therapy dramatically improves vision in child blindness
  • UCL: Childhood blindness therapy shows 'very promising' results
  • OT: From light perception to letters: patient’s vision improves following gene therapy
• December 2018 (re BEST1-ARB)
  • Scheie Eye Institute: Correcting macular degeneration with gene therapy: Canine models and human implications
• October 2018
  • Ophthotec: Ophthotech expands gene therapy pipeline in orphan retinal diseases with exclusive option for novel product candidates to treat Best vitelliform macular dystrophy (Best disease)
• August 2018 (re RHO-ADRP)
  • Penn News: Knockdown and replace: A gene therapy twofer to treat blindness
  • NIH NEI: Researchers find potential new gene therapy for blinding disease: Scientists funded by NIH preserve vision in dogs with a disease similar to retinitis pigmentosa in humans
  • Philadelphia Inquirer: Penn’s experimental gene therapy saves night vision in dogs, paving the way for human tests
  • UPI: New gene therapy treats inherited eye disorders in animal study
  • Ophthotec: Scientific data for novel adeno-associated virus gene therapy in rhodopsin-mediated autosomal dominant retinitis pigmentosa published in Proceedings of the National Academy of Sciences
• June 2018
  • Ophthotec: Ophthotech enters into gene therapy agreements with the University of Florida and the University of Pennsylvania
• April 2018
  • FFB: Groundbreaking research for inherited retinal diseases funded by Foundation Fighting Blindness to be showcased at ARVO
• March 2018 (re BEST1-ARB)
  • Penn News: New gene therapy corrects a form of inherited macular degeneration in canine model
  • GEN: Gene-therapy trials for inherited form of blindness could start in two years
  • Technology Networks: Best disease blindness bettered by gene therapy
  • BPoD: Blind spot
  • FFB: Best disease gene therapy advances toward clinical trial
• December 2017
  • ARVO: 2018 Achievement Award Recipients
• November 2017
  • ProQR: ProQR Doses First LCA 10 Patient in Clinical Trial of QR-110, ProQR’s Lead Program for Genetic Blindness
• August 2017
  • AGTC: Research Published in Molecular Therapy Identifies Optimal Gene-based Therapeutic Construct for X-Linked Retinitis Pigmentosa
• August 2016
  • Penn News: Penn: Blinding Disease in Canines and Humans Shares Causative Gene, Pathology
• December 2015
  • BPoD: Battling Blindness
• October 2015 (re RPGR-XLRP)
  • NEI News: Gene Therapy Staves Off Blindness from Retinitis Pigmentosa in Canine Model
  • Penn News: Penn Study Stops Vision Loss in Late-stage Canine X-linked Retinitis Pigmentosa
  • Scicasts: Study Stops Vision Loss in Late-stage Canine X-linked Retinitis Pigmentosa
  • FFB News: XLRP Gene Therapy Rescues Vision in Late-Stage Disease 
• May 2015 (re RPE65-LCA)
  • NEI News: Benefits for Leber Congenital Amaurosis Peak after One to Three Years, then Diminish
  • Penn News: Penn Study Indicates that Gene Therapy Efficacy for LCA is Dynamic: Improvement is Followed by Decline in Vision
  • Science Now: Gene Therapy for Blindness May Fade with Time
• Sept 2014
  • Nature News: Curing blindness: Vision quest
• March 2014
  • Penn News: Anatomical Discovery by Penn Team Gives Insight into Blinding Retinal Diseases
• January 2013
  • NEI News: New Findings Suggest Need for Combined Strategy in Treatment of Rare Form of Blindness
  • Penn News: Penn Study Sheds Light on the Complexity of Gene Therapy for Congenital Blindness
• January 2012
  • Penn News: Gene Therapy Research Team From Penn Vet and Scheie Eye Institute Cures Retinitis Pigmentosa in Dogs 
  • NEI News: Canine Study Puts Gene Therapy for X-linked Retinitis Pigmentosa in Sight
• August 2011
  • EurekAlert: Scientists Unravel the Cause of Rare Genetic Disease: Goldman-Favre Syndrome Explained
• December 2009
  • EyeWorld: One Year and Counting: Gene Therapy for Leber's Congenital Amaurosis
• September 2009
  • Philadelphia Inquirer: New Implants Allow a Ray of Hope for Blind Patients
• August 2009
  • EurekAlert: Vision Improvement after Gene Therapy Maintained at 1 Year for Inherited Retinal Blindness
  • MIT Technology Review: Gene Therapy Creates a New Fovea
  • EurekAlert: Vision Researchers See Unexpected Gain a Year into Blindness Trial
• September 2008
  • NEI News: Promising Results in Phase 1 Gene Therapy Trial for Blinding Disease
  • EurekAlert: Safety Study Indicates Gene Therapy for Blindness Improves Vision
• November 2007
  • EurekAlert: Gene Therapy Safety Trial for Childhood Blindness Under Way
• June 2007
  • EurekAlert: Penn Researchers Report that Gene Therapy Awakens the Brain Despite Blindness from Birth
• May 2006
  • EurekAlert: Gene Therapy for Chicken Model of Childhood Blindness
• April 2001
  • Science Daily: In Gene Therapy First, Scientists Restore Vision To Dogs Born Blind