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    Announcements

    Penn Vet’s Division of External Retinal Therapies is often in the news. Check back often to read the latest articles on our research.

    Fmri Activity

    2024

    Using fMRI, new vision study finds promising model for restoring cone function

    With the goal of developing novel gene and cell therapies that can restore daylight vision in people and in dogs, vision scientists from Penn Vet’s Division of ExpeRTs have developed a new fMRI protocol to measure non-invasively brain activity in the visual cortex that is triggered by cone photoreceptors. Using this approach they have demonstrated that a gene therapy for a form of day blindness restores cortical function.

    Aaas Logo

    2023

    William Beltran elected 2022 fellow of the American Association for the Advancement of Science (AAAS).

    The American Association for the Advancement of Science (AAAS) is an American international non-profit organization with the stated goals of promoting cooperation among scientists, defending scientific freedom, encouraging scientific responsibility, and supporting scientific education and science outreach for the betterment of all humanity.


    France Vet Academy

    2023

    Gustavo Aguirre and William Beltran elected to the French Academy of Veterinary Medicine

    Aguirre and Beltran were elected as lifetime members of the Académie Vétérinaire de France (AVF). The AVF provides guidance on scientific, technical, legal, historical and ethical fields in which the skills of the veterinarian are exercised, in particular those relating to animals, their diseases, their relations with humans and the environment, animal production, and veterinary public health. It contributes to the dissemination of scientific progress and the improvement of techniques relating to veterinary activities.

    William Beltran Endowed Professorship

    2022

    William Beltran Named Corinne R. and Henry Bower Professor of Ophthalmology at the University of Pennsylvania’s School of Veterinary Medicine

    Andrew M. Hoffman, DVM, DVSc, DACVIM, Gilbert S. Kahn Dean of the School of Veterinary Medicine at the University of Pennsylvania (Penn Vet) has named William Beltran, DVM, MSc, PhD, DECVO, an internationally recognized veterinary ophthalmologist, the Corinne R. and Henry Bower Professor of Ophthalmology.


    Replacing Photoreceptors

    2022

    Replacing dead photoreceptors

    As a potential new therapeutic approach aimed at restoring vision in blind people and animals that have lost all of their rods and cones due to retinal degeneration, vision scientists from Penn Vet’s Division of ExpeRTs, together with collaborators at the University of Wisconsin-Madison, the Children Hospital of Philadelphia, and the National Eye Institute, have shown long-term survival and integration of stem-cell derived photoreceptor precursor cells surgically implanted into canine eyes. These results pave the way to evaluating in dogs the functional benefit of this regenerative medicine strategy.


    Vector Screening Copy

    2022

    Seeing again in the dark

    Both people and dogs with congenital stationary night blindness (CSNB) have trouble perceiving objects in dim light. Vision scientists from Penn Vet’s Division of ExpeRTs, together with collaborators at the University of Pittsburgh, and University of California-Berkeley, have developed a novel gene therapy that restores night vision in dogs affected with this inherited condition. The results underscore the potential that a similar therapy might effectively treat CSNB in humans.

    Science Stem Cell Therapy

    2021

    Second round of funding from Fighting Blindness Canada to study new cell therapy for retinitis pigmentosa

    Vision scientists from Penn Vet’s Division of ExpeRTs, together with collaborators at the University of Wisconsin-Madison have been awarded CAD$725,000 from Fighting Blindness Canada’s Restore Vision 20/20 program to continue their ground-breaking research into cell replacement therapy for retinitis pigmentosa, an inherited retinal disease. The goal of this project is to test if a new stem cell therapy can improve vision in canine models of retinitis pigmentosa.


    Beltran Rna Sequencing

    2021

    Novel gene therapy platform speeds search for ways to cure blindness

    Vision scientists from Penn Vet’s Division of ExpeRTs, together with collaborators at the University of Pittsburgh, University of California-Berkeley, and Carnegie Mellon University have developed and used a new screening approach to identify novel viral vectors that can efficiently target numerous cell populations in the retina after a simple intraocular injection. This provides hope that some of these top-performing viral vectors can be used to deliver therapeutic genes to the retina and treat several blinding conditions in people and in dogs.

    Dr. Aguirre

    2020

    Gustavo Aguirre wins the Greenberg End Blindness 2020 Outstanding Achievement Prize

    Dr. Gustavo Aguirre, Professor of Ophthalmology and Medical Genetics, and Division of ExpeRTs investigator, was a co-recipient of the Sanford and Sue Greenberg Prize to End Blindness in recognition for his decades-long research with canine models of inherited retinal diseases that has led to several clinical trials for emerging therapies, including LUXTURNA


    National Academy of Medicine 2020 Awards

    2020

    William Beltran elected member of the National Academy of Medicine

    Dr. William Beltran, Professor of Ophthalmology and Director of the Division of ExpeRTS, was elected as a new member of the National Academy of Medicine. Founded 50 years ago, as the Institute of Medicine (IOM), the National Academy of Medicine (NAM) is one of the three academies that make up the National Academy of Sciences, Engineering, and Medicine (the National Academies) in the United States. The NAM’s mission is to improve health for all by advancing science, accelerating health equity, and providing independent, authoritative, and trusted advice on matters of science, technology, and health nationally and globally.

    Award Image

    2019

    Drs. Gustavo Aguirre and William Beltran win the Inventors of the Year Award

    Dr. Gustavo Aguirre, Professor of Ophthalmology and Medical Genetics, and Dr. William Beltran, Professor of Ophthalmology, were co-recipients of the Penn 2019 Inventors of the Year Award. This award recognizes their numerous contributions to the development of novel retinal gene therapies for inherited retinal degenerations.


    2019

    Mutation in LRIT3 underlies a canine model of stationary night blindness

    Investigators from the Division of ExpeRTs have identified a mutation in the LRIT3 gene responsible for congenital stationary night blindness (CSNB) in a new canine model. Identification of the molecular cause for canine CSNB that affects ON-bipolar cell function opens up possibilities to develop new approaches of gene therapy to target cell populations that are in the mid retina.


    2019

    Timing of Gene Therapy is critical to success

    An FDA-approved gene therapy for Leber congenital amaurosis, an inherited vision disorder with a childhood onset and progressive nature, has improved patients’ sight. But new research conducted by investigators from the Division of ExpeRTs on this blinding condition underscores the importance of defining the time-window for long-term therapeutic benefit.

    Leading the Fight Against Macular Degeneration

    A long-term collaboration between Division of ExpeRTs investigators from PennVet and Scheie Eye Institute to develop a gene therapy for a form of inherited macular degeneration caused by mutations in the BEST-1 gene was featured in Scheie Vision, the institute’s annual report.

    Scheie Vison cover

    Multidisciplinary team to develop stem cell-based approaches to restore vision

    Investigators from the Division of ExpeRTs are collaborating with Dr. David Gamm’s group (University of Wisconsin) to develop a stem-cell-based strategy to replace photoreceptors and treat blindness in dogs.

    An x ray of an eye.

    Dr. Gustavo Aguirre recognized as AAAS fellow

    Division of ExpeRTs investigator, Dr. Gustavo Aguirre (PennVet Professor of Ophthalmology and Medical Genetics) was recognized as AAAS fellow for his distinguished contributions to the field of inherited blindness.

    Two people with their arms wrapped arm and arm at an event.

    Knocking down Blindness: a novel gene therapy for autosomal dominant retinitis pigmentosa (adRP)

    Investigators from the Divisions of ExpeRTs together with collaborators from the University of Florida have developed a gene therapy capable of both knocking down and replacing rhodopsin, the most commonly mutated gene in patients affected with adRP. This approach was protective in dogs affected with a similar condition.

    Red and green graphics of gene therapy

    Dr. Gustavo Aguirre awarded the 2017 Proctor Medal

    Division of ExpeRTs investigator, Dr. Gustavo Aguirre (PennVet Professor of Ophthalmology and Medical Genetics) received the 2017 Proctor medal award in recognition for his long-term contribution to the field of retinal diseases and therapies. 

    A person being presented an award

    Inflammation during retinal degeneration

    A study conducted by Division of ExpeRTs investigators shows that signaling pathways associated with the innate immune system are activated during late stage disease in dogs with inherited retinal degenerations, and thus identifies potential molecular targets for development of novel therapies. 

    Green, orange, and red slide of retinal degenerations

    Identification of the underlying cause of a form of macular degeneration

    Division of ExpeRTs investigators from the School of Veterinary Medicine and Dental School identify BEST disease is caused by that loss of microvilli on the surface of RPE cells result in a loose connection with photoreceptors.

    Macular degeneration

    Before retinal cells die, they regenerate

    Division of ExpeRTs investigators have found that in at least three canine retinal diseases, photoreceptor cells proliferate before blindness takes hold. 

    Retinal Cell regenerate

    Dr. William Beltran awarded the ARVO Foundation’s 2016 Pfizer Ophthalmics Carl Camras Translational Research Award

    Director of the Division of ExpeRTs, Dr. William Beltran (PennVet Professor of Ophthalmology) received this award in recognition for his contribution at better understanding X-linked retinitis pigmentosa and developing a gene therapy for this early and severe form of inherited blindness. 

    Two people in front of a screen at an awards ceremony

    Dr. Gustavo Aguirre awarded the 2016 Louis Braille Award

    Division of ExpeRTs investigator, Dr. Gustavo Aguirre (PennVet Professor of Ophthalmology and Medical Genetics) was awarded the 2016 Louis Braille Award by the Associated Services for the Blind and Visually Impaired in recognitions for his innovative research and treatment of inherited blinding diseases. 


    Gene therapy for X-linked retinitis pigmentosa (XLRP) halts vision loss in late stage disease

    Investigators from the Division of ExpeRTs and Scheie Eye Institute that previously showed rescue of photoreceptors in dogs affected with a form of XLRP have now successfully demonstrated that this gene therapy is stable for more than 2 years and effective even when treatment is delivered after substantial loss of photoreceptors has occurred.

    Blue and green graph of tetinitis

    Cone Trouble

    Investigators from the Division of ExpeRTs identify two naturally-occurring mutations in dogs that cause day blindness by disrupting molecular channels involved in vision.

    Graphic of day blindness in dogs

    Anatomical Discovery by Penn Team Gives Insight into Blinding Retinal Diseases

    University of Pennsylvania vision scientists report that dogs, too, have an area of their retina that strongly resembles the human fovea. What’s more, this retinal region is susceptible to genetic blinding diseases in dogs just as it is in humans.


    Drs. Gustavo Aguirre and William Beltran awarded the Foundation Fighting Blindness Board of Directors Award

    Division of ExpeRTs investigators, Drs. Aguirre and Beltran were presented the FFB’s Board of Directors award at the VISION 2013 national conference in recognition for their research that is advancing treatments and cures for retinal degenerative diseases. 

    Four people standing with plaques.

    Penn Researchers take a first step toward a macular dystrophy gene therapy

    Investigators from the Division of ExpeRTs, identify an optimal viral vector serotype for future gene therapy studies aimed restoring at a normal copy of the gene (BEST1) that is mutated in an inherited form of macular dystrophy called Best disease.

    macular-dystrophy-gene-therapy


    Dr. Gustavo Aguirre winner of the 2013 International Canine Health Award

    Dr. Gustavo Aguirre, Professor of Ophthalmology & Medical Genetics received the International Canine Health Award from the Kennel Club Charitable Trust for his contribution to the advancement of research into canine eye diseases. The Kennel Club Charitable Trust is the UK’s largest organization dedicated to protecting and promoting the health and welfare of all dogs. International Canine Health Awards are underwritten by a major gift from Vernon and Shirley Hill.

    Two people standing with a plaque

    First gene therapy for a common form of X-linked retinitis pigmentosa shows promise in dogs

    A consortium of investigators from PennVet’s Division of ExpeRTs, the Scheie Eye Institute, and the University of Florida have developed a gene therapy for the most common cause of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. This novel therapy prevents disease onset and halts ongoing photoreceptor death in dogs carrying two distinct mutations in this same gene.

    Illustration of x-linked retinitis

    Dr. Gustavo Aguirre elected member of the Institute of Medicine

    Dr. Gustavo Aguirre, Professor of Ophthalmology & Medical Genetics was elected as a new member of the Institute of Medicine. Established in 1970 by the National Academy of Sciences, IOM has become recognized as a national resource for independent, scientifically informed analysis and recommendations on health issues. With their election, members make a commitment to volunteer their service on IOM committees, boards, and other activities.

    Gustavo