Evidence map›Paper›PMID 39890493›Full record

ReviewTrends in molecular medicine2025

Adeno-associated viral vector targeted evolution for neurofibromatosis gene delivery.

Brett Henshey, Ana Carneiro, Kecheng Lei, David Schaffer, Nicholas M Boulis

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Trends in molecular medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Neurofibromin 1 (International journal of molecular sciences · 2026
    Article
  2. Review
  3. 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

5 authors.

Brett HensheyDepartment of Neurosurgery, Emory University School of Medicine, Atlanta, GA, USA.
Ana CarneiroDepartment of Chemical and Biomolecular Engineering, University of California, Berkeley, Berkeley, CA 94720, USA.
Kecheng LeiDepartment of Neurosurgery, Emory University School of Medicine, Atlanta, GA, USA. Electronic address: kecheng.lei@emory.edu.
David SchafferDepartment of Chemical and Biomolecular Engineering, University of California, Berkeley, Berkeley, CA 94720, USA; Department of Bioengineering, University of California, Berkeley, Berkeley, CA 94720, USA; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA 94720, USA; California Institute for Quantitative Biosciences (QB3), University of California, Berkeley, Berkeley, CA 94720, USA; Helen Wills Neuroscience Institute, University of California, Berkeley, Berkeley, CA 94720, USA. Electronic address: schaffer@berkeley.edu.
Nicholas M BoulisDepartment of Neurosurgery, Emory University School of Medicine, Atlanta, GA, USA; Department of Biomedical Engineering, Georgia Institute of Technology, Atlanta, GA, USA. Electronic address: nboulis@emory.edu.

Funding

Implementing a Maternal health and PRegnancy Outcomes Vision for Everyone (IMPROVE)UL1TR002378 · NCATS · EMORY UNIVERSITY · PI Andres J Garcia, Elizabeth O. Ofili · 2017 to 2026
$92.1M
Institutional Career Development CoreKL2TR002381 · NCATS · EMORY UNIVERSITY · PI HENRY M BLUMBERG, Anandi Nayan Sheth · 2017 to 2026
$14.1M
Neuro HPA Project 1U54AG062334 · NIA · EMORY UNIVERSITY · PI Ighovwerha Ofotokun · 2018 to 2026
$12.2M
The 'Career MODE' Program: Careers through Mentoring and training in Omics and Data for Early-stage investigatorsR25GM143298 · NIGMS · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI IONITA, IULIANA, RE, DIANE BERENGERE · 2021 to 2025
$2.3M
NCATS NIH HHS KL2 TR002381NCATS NIH HHS UL1 TR002378NIA NIH HHS U54 AG062334NIGMS NIH HHS R25 GM143298
6 · The paper itself

Abstract

Neurofibromatosis type 1 (NF1) is an inherited genetic disease resulting from pathogenic mutations in NF1 that drive tumor formation along peripheral nerves, leading to many functional consequences. Tumor removal or treatment often results in regrowth and/or nerve damage. Addressing NF1 pathogenic variations at the cellular level through gene therapy holds great potential for long-term treatment of patients with NF1. Adeno-associated viruses (AAVs) are broadly used gene delivery vehicles for gene therapies because of their low pathogenicity, ability to transduce nondividing cells, and potential for long-term gene expression. This article explores the landscape of AAV-mediated gene delivery strategies for NF1, discusses the challenges of efficient delivery to relevant cell types, and highlights the progress in vector design strategies.

Indexed as

DependovirusGenetic TherapyGenetic VectorsGene Transfer TechniquesNeurofibromatosis 1AnimalsHumansadeno-associated virus (AAV)capsid engineeringgene therapyneurofibromatosis type 1 (NF1)peripheral nervous system (PNS)

Identifiers

PMID39890493
PMCPMC11985305

What OpenQuestion holds

Textmetadata
LicenceTDM
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.