Evidence map›Paper›PMID 40862658›Full record

ReviewTranslational vision science & technology2025

Addressing Challenges in Developing Treatments for Inherited Retinal Diseases: Recommendations From the Third Monaciano Symposium.

Debra A Thompson, K Thiran Jayasundera, Oleg Alekseev, Robin R Ali, Alessia Amato, Vadim Y Arshavsky, Isabelle S Audo, Alberto Auricchio, James W B Bainbridge, Eyal Banin and 31 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Translational vision science & technology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Review
  2. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

41 authors.

Debra A ThompsonKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
K Thiran JayasunderaKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
Oleg AlekseevDepartment of Ophthalmology, Duke University School of Medicine, Durham, NC, USA.
Robin R AliCentre for Gene Therapy and Regenerative Medicine, King's College London, London, UK.
Alessia AmatoDepartment of Ophthalmology, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
Vadim Y ArshavskyDepartment of Ophthalmology, Duke University School of Medicine, Durham, NC, USA.
Isabelle S AudoInstitut de la Vision, Sorbonne Université, INSERM, CNRS, Paris, France.
Alberto AuricchioTelethon Institute of Genetics and Medicine, Naples, Italy.
James W B BainbridgeInstitute of Ophthalmology, University College London, London, UK.
Eyal BaninCenter for Retinal and Macular Degenerations, Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Ein-Karem, Jerusalem, Israel.
Cagri G BesirliKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
David G BirchRetina Foundation of the Southwest, Dallas, TX, USA.
Kari E BranhamKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
Artur V CideciyanCenter for Hereditary Retinal Degenerations, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA.
Stephen P DaigerHuman Genetics Center, Epidemiology Department, School of Public Health, University of Texas Health Science Center-Houston, Houston, TX, USA.
Jacque L DuncanWayne and Gladys Valley Center for Vision, Department of Ophthalmology, University of California-San Francisco, San Francisco, CA, USA.
Abigail T FahimKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
John G FlanneryHelen Wills Neuroscience Institute, University of California-Berkeley, Berkeley, CA, USA.
Roberto GattegnaRetina Service, Israelitic Hospital, Roma, Italy.
John R HeckenlivelyKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
Elise HéonDepartment of Ophthalmology and Vision Sciences, Hospital for Sick Children, Toronto, Ontario, Canada.
Alessandro IannacconeCell and Gene Therapy, Biopharma and Ophthalmology Division, Astellas Pharma US, Northbrook, IL, USA.
Naheed W KhanKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
Samer KhatebCenter for Retinal and Macular Degenerations, Department of Ophthalmology, Hadassah-Hebrew University Medical Center, Ein-Karem, Jerusalem, Israel.
Henry J KlassenGavin Herbert Eye Institute, Stem Cell Research Center, University of California-Irvine, Irvine, CA, USA.
Bart P LeroyDepartment of Ophthalmology & Center for Medical Genetics, Ghent University Hospital & Ghent University, Ghent, Belgium.
Dario MarangoniDepartment of Medicine, Surgery and Health Sciences, Ophthalmology Clinic, University of Trieste, Trieste, Italy.
Michel MichaelidesInstitute of Ophthalmology, University College London, London, UK.
David C MuschKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
Mark E PennesiRetina Foundation of the Southwest, Dallas, TX, USA.
Simon M Petersen-JonesDepartment of Small Animal Clinical Sciences, College of Veterinary Medicine, Michigan State University, East Lansing, MI, USA.
Eric A PierceOcular Genomics Institute, Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston, MA, USA.
Rajesh C RaoKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
José-Alain SahelInstitut de la Vision, Sorbonne Université, INSERM, CNRS, Paris, France.
Paul A SievingCenter for Ocular Regenerative Therapy, Department of Ophthalmology, University of California-Davis School of Medicine, Sacramento, CA, USA.
Enrica StrettoiCNR Neuroscience Institute, Area della Ricerca, Pisa, Italy.
Cameron R StrongDepartment of Physical Medicine and Rehabilitation, University of Washington, Seattle, WA, USA.
Thomas J WubbenKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
Paul YangCasey Eye Institute, Oregon Health Science Center, Portland, OR, USA.
David N ZacksKellogg Eye Center, Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor, MI, USA.
Monaciano Consortium

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Over the past decade, efforts focused on developing genetic therapies for inherited retinal diseases have advanced steadily to clinical trials and the development of a treatment, fueling optimism for the potential of precision medicines to provide safe and effective therapies for these rare conditions. Although several ongoing programs remain poised for success, numerous challenges have negatively impacted the ability to obtain regulatory approvals. The present position paper briefly summarizes recent advances and challenges in developing therapeutics for inherited retinal diseases, and presents a set of recommendations for moving the field forward. The priorities identified are discussed in terms of progress made and future needs, focusing on areas including patient support, disease mechanisms, outcome measures, and therapy approvals. A key point is the potential value of restructuring collaborative interactions into broadly resourced enterprises that are comprehensive in scope across critical areas of science, business, and medicine.

Indexed as

Genetic TherapyRetinal DiseasesHumans

Identifiers

PMID40862658
PMCPMC12395841

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.