Press

September 2024 (re GUCY2D-LCA1 gene therapy, * original reporting)
- MedicalNewsToday (*): Gene therapy for vision loss: Ongoing challenges
- WHYY (*): Penn researchers develop new gene therapy for previously untreatable eye disorder
- SciTechDaily: Seeing Is believing: The gene therapy breakthrough that’s giving sight to the blind
- MedPage Today (*): Gene therapy shows promise in rare eye disease that causes early blindness
- Medscape Medical News (*): Gene therapy shown to improve vision in congenital blindness
- STAT News (*): Q&A: Atsena Therapeutics’ gene therapy for inherited blindness shows promise
- European Pharmaceutical Review: Gene therapy trial facilitates major vision improvement
- Precision Medicine Online: Promising 12-month data on Atsena's LCA1 gene therapy bolsters case of pivotal trial
- Insight News: Gene therapy trial brings dramatic improvement in eyesight for patients
- HealthDay: Gene therapy reverses a rare cause of vision loss
- Correio Braziliense (*): Terapia genética melhora 10 mil vezes a visão de pacientes de doença rara [Gene therapy improves vision of rare disease patients 10,000 times ]
- US News & World Report: Gene Therapy Reverses a Rare Cause of Vision Loss
- UF News: Gene therapy restores vision in first-ever trial for rare, inherited blindness
- Atsena: Atsena therapeutics announces 12-month safety and efficacy data from phase I/II clinical trial of ATSN-101 in LCA1 published in The Lancet
- Penn Today: Gene therapy trial sees massive improvement in vision
- Penn Medicine News: 100x improvement in sight seen after gene therapy trial
August 2024 (re LCA5 gene therapy)
- Opus Genetics: Opus genetics receives rare pediatric disease designation from the U.S. FDA for ocular gene therapy OPGx-LCA5 to treat rare inherited retinal disease LCA5
May-June 2024 (re CEP290 gene editing)
- CHOP Cornerstone Blog: CHOP-Penn team tests first in-body CRISPR therapy to treat blindness
- Penn Medicine News: Gene editing restores some sight in pair of children treated for blindness
- CHOP: Two CHOP patients with an inherited blindness successfully treated with gene editing
- The Philadelphia Inquirer: CHOP restored eyesight for two children with one injection. It’s one more step for gene editing using CRISPR.
- CBS News: CHOP helps restore vision in 2 children using experimental gene editing treatment
- Science: Gene editing improves some blind patients’ vision
- US News and World Report: Gene therapy improves vision in people with inherited blindness
- CNN: Experimental gene therapy restores some vision in patients with inherited blindness
- Ophthalmology Times: CRISPR gene editing leads to improvements in vision for people with inherited blindness
March 2024 (re DHDDS)
- UAB News: ALG6 acts as a modifier gene in the inherited genetic eye disease retinitis pigmentosa 59
- NEI Research News: ALG6 acts as a modifier gene in the inherited genetic eye disease retinitis pigmentosa 59
September-November 2023 (re LCA5 gene therapy)
- Yahoo Finance: New preliminary clinical data on potential of Opus AAV-based gene therapy for rare inherited retinal disease to be presented at the American Academy of Ophthalmology
- GlobeNewswire: Opus Genetics announces first patient dosed in phase 1/2 trial of gene therapy OPGx-LCA5 in patients with rare inherited retinal disease LCA5
- FFB Eye on the Cure Research News: First patient dosed in LCA5 gene therapy clinical trial launched by Opus Genetics
August 2023
- FFB Eye on the Cure Podcast: Host Ben Shaberman interviews Artur Cideciyan about his experience as an investigator in several prominent clinical trials.
May-July 2023 (re NPHP5 selected for gene therapy)
- The Philadelphia Inquirer: These 8 diseases are so rare that drug firms haven’t tried treating them with gene therapy. A $97 million program aims to help.
- Pharma News Intelligence: FNIH enhances rare disease research by adding 8 diseases to BGTC
- USA Today: A new research effort takes aim at 8 rare diseases. It could revolutionize many more.
- FNIH: The foundation for the National Institutes of Health announces selection of eight rare diseases for the Bespoke Gene Therapy Consortium clinical trial portfolio
April 2023 (re Hellen Keller Prize)
- Penn Today: Four from Penn awarded Helen Keller prize for vision research
October 2022 - January 2023 (re GUCY2D-LCA)
- Drug Discovery News: A new gene therapy for inherited blindness
- Live Science: New gene therapy restores night vision of people with inherited eye disorder
- US News & World Report: Gene therapy helps those with rare form of blindness see better at night
- Ophthalmology Times: Study: Gene therapy rapidly improves night vision in adults with congenital blindness
- Interesting Engineering: A new gene therapy method improves night vision in adults with congenital blindness, scientists suggest
- Penn Today: Gene therapy rapidly improves night vision in adults with congenital blindness.
- Penn Medicine: Gene therapy rapidly improves night vision in adults with congenital blindness, Penn study finds
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