Evidence map›Paper›PMID 42597498›Full record

ArticleMolecular therapy. Nucleic acids2026

Development of an antisense oligonucleotide targeting EFEMP1 in models relevant to macular degeneration.

William P Miller, Said Arevalo-Alquichire, Paula Perez-Corredor, Rose Lin, Anil Upreti, Jeysson Sanchez-Suarez, Tim E Vanderleest, Claudia Marino, Donovan Spencer, Matthew Scribner and 17 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

27 authors.

William P MillerSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Said Arevalo-AlquichireSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Paula Perez-CorredorSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Rose LinSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Anil UpretiSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Jeysson Sanchez-SuarezSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Tim E VanderleestSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Claudia MarinoSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Donovan SpencerSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Matthew ScribnerSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Jebgy VargasSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Andres Muriel-TorresSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Xinyao HuSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Yvonne Adu-RutledgeSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Bryan A KaplanSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Karim W BarakeSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Audrey L GunawanSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Aruvi VijikumarSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Reeya ShresthaSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Anton LennikovSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Leo A KimMass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Joseph F Arboleda-VelasquezSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.
Andrew PetersonAldebaran Therapeutics, Waltham, MA, USA.
Sudhir AgrawalAldebaran Therapeutics, Waltham, MA, USA.
Vinod VathipadiekalAlloy Therapeutics, Waltham, MA, USA.
Carlos LoyaAldebaran Therapeutics, Waltham, MA, USA.
Michael O'HareSchepens Eye Research Institute of Mass Eye and Ear and the Department of Ophthalmology at Harvard Medical School, Boston, MA 02114, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Antisense oligonucleotides (ASOs) are RNA-targeting therapeutics with broad potential for genetically defined and complex ocular diseases. The eye is particularly well suited for ASO delivery because of its compartmentalized anatomy, accessibility, and capacity for sustained intraocular drug retention. Autosomal dominant drusen (ADD), an inherited retinal dystrophy characterized by early drusen formation and secondary choroidal neovascularization (CNV), is caused by the EFEMP1 R345W mutation. Mutant EFEMP1 accumulates within the retinal pigment epithelium (RPE) and extracellular matrix, contributing to disease pathology. EFEMP1 is also elevated in the serum and RPE/choroid of patients with age-related macular degeneration (AMD), although its functional role in AMD remains incompletely defined. Here, we developed a biallelic EFEMP1-targeted ASO that achieved potent knockdown and modulated disease-relevant pathways in iPSC-derived RPE, human microvascular retinal endothelial cells (HMRECs),

Indexed as

age-related macular degenerationangiogenesisantisense oligonucleotidesautsomal dominant drusenEFEMP1MT: oligonucleotides, therapies and applications

Identifiers

PMID42597498
PMCPMC13469845

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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.