Evidence map›Paper›PMID 39563031›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

Longitudinal imaging of therapeutic enzyme expression after gene therapy for Fabry disease using positron emission tomography and the radiotracer [

Charalambos Kaittanis, Tyler Teceno, Ashley Knight, Yoann Petibon, Phil Sandoval, Lawrence Cohen, Shin Hye Ahn, Anthony P Belanger, Louise M Clark, Quang-De Nguyen and 11 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed, 1 pooled it
–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

6 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Next-generation molecular imaging of cardiac fibrosis.The international journal of cardiovascular imaging · 2026
    Review
  3. Review
  4. Review
  5. Where did my vector go and how well did it function? PET biodistribution studies of viral gene therapy vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  6. Review
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

21 authors.

Charalambos KaittanisTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Tyler TecenoTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Ashley KnightTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Yoann PetibonTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Phil SandovalTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Lawrence CohenTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Shin Hye AhnMolecular Cancer Imaging Facility, Dana-Farber Cancer Institute, Boston, MA 02215, USA.
Anthony P BelangerMolecular Cancer Imaging Facility, Dana-Farber Cancer Institute, Boston, MA 02215, USA.
Louise M ClarkLurie Family Imaging Center, Dana-Farber Cancer Institute, Boston, MA 02215, USA.
Quang-De NguyenLurie Family Imaging Center, Dana-Farber Cancer Institute, Boston, MA 02215, USA.
Wanida RuangsirilukTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Shreya MukherjiTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Cristian C ConstantinescuInvicro, New Haven, CT 06510, USA.
Amy Llopis AmentaInvicro, New Haven, CT 06510, USA.
Sarav NarayananTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Mugdha DeshpandeTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Rizwana IslamTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Shipeng YuanTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Paul McQuadeTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Christopher T WinkelmannTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA.
Talakad G LohithTakeda Pharmaceutical Company Limited, Cambridge, MA 02142, USA. Electronic address: talakad.lohith@takeda.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Longitudinal, non-invasive, in vivo monitoring of therapeutic gene expression is an unmet need for gene therapy (GT). Positron emission tomography (PET) radiotracers designed to bind to therapeutic proteins may provide a sensitive imaging platform to guide treatment response and dose optimization in GT. Herein, we evaluate a novel PET tracer ([

Indexed as

alpha-GalactosidaseFabry DiseaseFluorine RadioisotopesGenetic TherapyPositron-Emission TomographyAnimalsDependovirusDisease Models, AnimalGene ExpressionGenetic VectorsHumansMiceMice, KnockoutRadiopharmaceuticalsTissue Distributionalpha-GalactosidaseFluorine RadioisotopesRadiopharmaceuticalsadeno-associated virusenzyme replacement therapyFabry diseasegene expressiongene therapyheartimaging biomarkermolecular genetic imagingPET imagingα-galactosidase A

Identifiers

PMID39563031
PMCPMC12432858

What OpenQuestion holds

Textmetadata
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.