Evidence map›Paper›PMID 37601414›Full record

ArticleMolecular therapy. Methods & clinical development2023

Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.

Manuela Corti, Barry J Byrne, Dominic J Gessler, Grace Thompson, Samantha Norman, Jenna Lammers, Kirsten E Coleman, Cristina Liberati, Melissa E Elder, Maria L Escolar and 8 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed
4.2field-weighted citation impact, top 5% 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

24 citing papers in PubMed, 27 citations in OpenAlex.

  1. Trial
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  8. Article
  9. Genetic therapies for neurological diseases.Pharmacological reviews · 2026
    Review
  10. Article
  11. Current clinical applications of AAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  12. The curious case of AAV immunology.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  13. Review
  14. Review
  15. Adeno-Associated Viral Vectors in the Treatment of Epilepsy.International journal of molecular sciences · 2024
    Review
  16. Review
  17. Review
  18. Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024
    Review
  19. Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024
    Review
  20. 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

18 authors at 6 institutions in 1 country.

Manuela CortiPowell Gene Therapy Center, Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA.
Barry J ByrnePowell Gene Therapy Center, Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA.
Dominic J GesslerHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
Grace ThompsonDepartment of Pediatric Surgery, College of Medicine, University of Florida, Gainesville, FL, USA.
Samantha NormanPowell Gene Therapy Center, Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA.
Jenna LammersPowell Gene Therapy Center, Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA.
Kirsten E ColemanPowell Gene Therapy Center, Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA.
Cristina LiberatiPowell Gene Therapy Center, Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA.
Melissa E ElderPowell Gene Therapy Center, Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA.
Maria L EscolarDepartment of Pediatrics, School of Medicine, University of Pittsburgh, Pittsburgh, PA, USA.
Ibrahim S TunaDepartment of Radiology, College of Medicine, University of Florida, Gainesville, FL, USA.
Clementina MesarosPenn Medicine/Children's Hospital of Philadelphia Center of Excellence in Friedreich's Ataxia, University of Pennsylvania, Philadelphia, PA, USA.
Gary I KleinerDepartment of Pediatrics, Miller School of Medicine, University of Miami, Miami, FL, USA.
Deborah S BarbouthDivision of Clinical and Translational Genetics, Department of Human Genetics, Miller School of Medicine, University of Miami, Miami, FL, USA.
Heather L Gray-EdwardsHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
Nathalie ClementPowell Gene Therapy Center, Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA.
Brian D CleaverPowell Gene Therapy Center, Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA.
Guangping GaoHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
University of Florida · USUniversity of Massachusetts Chan Medical School · USUniversity of Miami · USChildren's Hospital of Philadelphia · USUniversity of Florida Health · USUniversity of Pittsburgh · US

Funding

Translational Research Support CoreP30ES013508 · NIEHS · UNIVERSITY OF PENNSYLVANIA · PI Trevor M Penning · 2006 to 2026
$35.3M
Oligodendrocyte-focused rAAV gene therapy strategies for Canavan disease and LeukodystrophiesR01NS076991 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Guangping Gao · 2012 to 2026
$5.7M
NINDS NIH HHS R01 NS076991
6 · The paper itself

Abstract

Gene replacement therapy is a rational therapeutic strategy and clinical intervention for neurodegenerative disorders like Canavan disease, a leukodystrophy caused by biallelic mutations in the aspartoacylase (

Indexed as

AAVCanavan diseasedual route of administrationimmune modulation

Identifiers

PMID37601414
PMCPMC10432950
OpenAlexW4381252610

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.