Evidence map›Paper›PMID 40932991›Full record

ArticleMolecular therapy. Methods & clinical development2025

AAV hamartin gene therapy in a stochastic, cerebral mouse model of tuberous sclerosis type 1.

Edwina Abou Haidar, Shilpa Prabhakar, Alexandra L Geffrey, Mohammed Mahamdeh, Tomeh Tomeh, Koen Breyne, Elie Roumieh, Mia Gurevich, Roy J Soberman, Gregory R Wojtkiewicz and 4 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

14 authors.

Edwina Abou HaidarMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02129, USA.
Shilpa PrabhakarMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02129, USA.
Alexandra L GeffreyDepartment of Neurology, Massachusetts General Hospital, Boston, MA 02114, and Harvard Medical School, Boston, MA 02115, USA.
Mohammed MahamdehCardiovascular Research Center, Cardiology Division, Department of Medicine, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02129, USA.
Tomeh TomehMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02129, USA.
Koen BreyneMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02129, USA.
Elie RoumiehMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02129, USA.
Mia GurevichMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02129, USA.
Roy J SobermanMass General Brigham Center of Excellence for Molecular Imaging, Boston, MA 02129, USA.
Gregory R WojtkiewiczCenter for Systems Biology, Massachusetts General Hospital, Boston, MA 02114, USA.
John W ChenCenter for Systems Biology and Department of Radiology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02114, USA.
Justin Seungkyu HanDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, CCXDP, Boston, MA 02129, USA.
Anat Stemmer-RachamimovDepartment of Pathology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02114, USA.
Xandra O BreakefieldDepartment of Neurology, Department of Radiology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02114, USA.

Funding

Boosting IL-12-induced anti-glioblastoma activity via immunotherapeutic extracellular vesicles.K22CA282019 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI Koen Breyne · 2024 to 2026
$482k
NCI NIH HHS K22 CA282019
6 · The paper itself

Abstract

Tuberous sclerosis complex (TSC) is a dominantly inherited disease in which most individuals are born with one defective allele encoding for either hamartin (TSC1) or tuberin (TSC2), with a somatic loss of the other allele leading to abnormal neurodevelopment and upregulation of cell growth in susceptible tissues. Ninety percent of affected individuals have brain involvement, including epilepsy, cognitive impairment, autism, and/or sleep disorders. In the stochastic, cerebral mouse model of Tsc1, loss of function of hamartin is induced in the CNS by injection of an adeno-associated virus (AAV) vector encoding Cre recombinase into the cerebral ventricles of homozygous Tsc1

Indexed as

AAV9braingene replacementmicroglia activationmyelinationTSC1tuberous sclerosis complex

Identifiers

PMID40932991
PMCPMC12419009

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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.