Evidence map›Paper›PMID 38397064›Full record

ReviewInternational journal of molecular sciences2024

A New Generation of Gene Therapies as the Future of Wet AMD Treatment.

Janusz Blasiak, Elzbieta Pawlowska, Justyna Ciupińska, Marcin Derwich, Joanna Szczepanska, Kai Kaarniranta

Open access · goldAbstract readReview
In one paragraph

Review in International journal of molecular sciences, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
5.4field-weighted citation impact, top 3% of its field
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

14 citing papers in PubMed, 23 citations in OpenAlex.

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  10. EvaluatingMedicina (Kaunas, Lithuania) · 2024
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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

6 authors at 3 institutions in 2 countries.

Janusz BlasiakFaculty of Medicine, Collegium Medicum, Mazovian Academy in Plock, 09-402 Plock, Poland.ORCID 0000-0001-9539-9584
Elzbieta PawlowskaDepartment of Pediatric Dentistry, Medical University of Lodz, 92-217 Lodz, Poland.ORCID 0000-0002-5373-4783
Justyna CiupińskaClinical Department of Infectious Diseases and Hepatology, H. Bieganski Hospital, 91-347 Lodz, Poland.
Marcin DerwichDepartment of Pediatric Dentistry, Medical University of Lodz, 92-217 Lodz, Poland.ORCID 0000-0002-7203-271X
Joanna SzczepanskaDepartment of Pediatric Dentistry, Medical University of Lodz, 92-217 Lodz, Poland.
Kai KaarnirantaDepartment of Ophthalmology, University of Eastern Finland, 70210 Kuopio, Finland.ORCID 0000-0003-2600-8679
Medical University of Lodz · PLGrochowski Hospital · PLUniversity of Eastern Finland · FI

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Age-related macular degeneration (AMD) is an eye disease and the most common cause of vision loss in the Western World. In its advanced stage, AMD occurs in two clinically distinguished forms, dry and wet, but only wet AMD is treatable. However, the treatment based on repeated injections with vascular endothelial growth factor A (VEGFA) antagonists may at best stop the disease progression and prevent or delay vision loss but without an improvement of visual dysfunction. Moreover, it is a serious mental and financial burden for patients and may be linked with some complications. The recent first success of intravitreal gene therapy with ADVM-022, which transformed retinal cells to continuous production of aflibercept, a VEGF antagonist, after a single injection, has opened a revolutionary perspective in wet AMD treatment. Promising results obtained so far in other ongoing clinical trials support this perspective. In this narrative/hypothesis review, we present basic information on wet AMD pathogenesis and treatment, the concept of gene therapy in retinal diseases, update evidence on completed and ongoing clinical trials with gene therapy for wet AMD, and perspectives on the progress to the clinic of "one and done" therapy for wet AMD to replace a lifetime of injections. Gene editing targeting the

Indexed as

Vascular Endothelial Growth Factor AWet Macular DegenerationAngiogenesis InhibitorsGenetic TherapyHumansAngiogenesis InhibitorsVascular Endothelial Growth Factor Aage-related macular degenerationAMDanti-VEGF therapyclinical trialsgene editinggene therapyvascular endothelial growth factorVEGF

Identifiers

PMID38397064
PMCPMC10888617
OpenAlexW4391946786

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.