ArticleMolecular therapy. Methods & clinical development2021
Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis.
Article in Molecular therapy. Methods & clinical development, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
What it found
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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.
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.
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Who cites it
11 citing papers in PubMed, 16 citations in OpenAlex.
- MRI detection of senescent cells in porcine knee joints with a β-galactosidase responsive Gd-chelate.Npj imaging · 2025Article
- MR Imaging Reveals Dynamic Aggregation of Multivalent Glycoconjugates in Aqueous Solution.Inorganic chemistry · 2024Article
- The role of responsive MRI probes in the past and the future of molecular imaging.Chemical science · 2024Review
- Musculoskeletal imaging of senescence.Skeletal radiology · 2024Review
- Stimuli-responsive linkers and their application in molecular imaging.Exploration (Beijing, China) · 2024Review
- Imaging of adeno-associated viral capsids for purposes of gene editing using CEST NMR/MRI.Magnetic resonance in medicine · 2024Article
- A Dual-Gene Reporter-Amplifier Architecture for Enhancing the Sensitivity of Molecular MRI by Water Exchange.Chembiochem : a European journal of chemical biology · 2024Article
- A dual-gene reporter-amplifier architecture for enhancing the sensitivity of molecular MRI by water exchange.bioRxiv : the preprint server for biology · 2024Article
- Review
- AAV-based in vivo gene therapy for neurological disorders.Nature reviews. Drug discovery · 2023Review
- Molecular Engineering of Self-Immolative Bioresponsive MR Probes.Journal of the American Chemical Society · 2023Article
Corrections and comments
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Authors and funding
17 authors at 4 institutions in 1 country.
Funding
Abstract
Transformative results of adeno-associated virus (AAV) gene therapy in patients with spinal muscular atrophy and Leber's congenital amaurosis led to approval of the first two AAV products in the United States to treat these diseases. These extraordinary results led to a dramatic increase in the number and type of AAV gene-therapy programs. However, the field lacks non-invasive means to assess levels and duration of therapeutic protein function in patients. Here, we describe a new magnetic resonance imaging (MRI) technology for real-time reporting of gene-therapy products in the living animal in the form of an MRI probe that is activated in the presence of therapeutic protein expression. For the first time, we show reliable tracking of enzyme expression after a now in-human clinical trial AAV gene therapy (ClinicalTrials.gov: NTC03952637) encoding lysosomal acid beta-galactosidase (βgal) using a self-immolative βgal-responsive MRI probe. MRI enhancement in AAV-treated enzyme-deficient mice (GLB-1
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Registered trials
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.