Evidence map›Paper›PMID 29858078›Full record

ArticleMolecular therapy. Nucleic acids2018

CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer's Disease.

Bence György, Camilla Lööv, Mikołaj P Zaborowski, Shuko Takeda, Benjamin P Kleinstiver, Caitlin Commins, Ksenia Kastanenka, Dakai Mu, Adrienn Volak, Vilmantas Giedraitis and 6 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 103 papers.

0numbers the graph read from it
0cells of the map it votes in
103citing 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

103 citing papers in PubMed.

  1. Safety considerations of gene-based therapies for Alzheimer's disease.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2026
    Review
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  6. Review
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  9. Genetic therapies for neurological diseases.Pharmacological reviews · 2026
    Review
  10. Review
  11. Article
  12. Article
  13. Review
  14. Article
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  17. Article
  18. Review
  19. Article
  20. Review

43 more citing papers are in PubMed but not listed here.

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

16 authors.

Bence GyörgyDepartments of Neurology and Radiology, Massachusetts General Hospital and Center for NeuroDiscovery, Harvard Medical School, Boston, MA, USA; Department of Neurobiology, Harvard Medical School, 220 Longwood Avenue, Boston, MA 02115, USA.
Camilla LöövDepartments of Neurology and Radiology, Massachusetts General Hospital and Alzheimer's Disease Research Center, Harvard Medical School, Boston, MA, USA.
Mikołaj P ZaborowskiDepartments of Neurology and Radiology, Massachusetts General Hospital and Center for NeuroDiscovery, Harvard Medical School, Boston, MA, USA; Department of Gynecology, Obstetrics and Gynecologic Oncology, Division of Gynecologic Oncology, Poznan University of Medical Sciences, 60-535 Poznan, Poland.
Shuko TakedaDepartments of Neurology and Radiology, Massachusetts General Hospital and Alzheimer's Disease Research Center, Harvard Medical School, Boston, MA, USA.
Benjamin P KleinstiverMolecular Pathology Unit, Massachusetts General Hospital, Charlestown, MA, USA; Center for Cancer Research, Massachusetts General Hospital, Charlestown, MA, USA; Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, MA, USA; Department of Pathology, Harvard Medical School, Boston, MA, USA.
Caitlin ComminsDepartments of Neurology and Radiology, Massachusetts General Hospital and Alzheimer's Disease Research Center, Harvard Medical School, Boston, MA, USA.
Ksenia KastanenkaDepartments of Neurology and Radiology, Massachusetts General Hospital and Alzheimer's Disease Research Center, Harvard Medical School, Boston, MA, USA.
Dakai MuDepartments of Neurology and Radiology, Massachusetts General Hospital and Center for NeuroDiscovery, Harvard Medical School, Boston, MA, USA.
Adrienn VolakDepartments of Neurology and Radiology, Massachusetts General Hospital and Center for NeuroDiscovery, Harvard Medical School, Boston, MA, USA.
Vilmantas GiedraitisDepartment of Public Health and Caring Sciences, Geriatrics, Rudbeck Laboratory, Uppsala University, Uppsala, Sweden.
Lars LannfeltDepartment of Public Health and Caring Sciences, Geriatrics, Rudbeck Laboratory, Uppsala University, Uppsala, Sweden.
Casey A MaguireDepartments of Neurology and Radiology, Massachusetts General Hospital and Center for NeuroDiscovery, Harvard Medical School, Boston, MA, USA.
J Keith JoungMolecular Pathology Unit, Massachusetts General Hospital, Charlestown, MA, USA; Center for Cancer Research, Massachusetts General Hospital, Charlestown, MA, USA; Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, MA, USA; Department of Pathology, Harvard Medical School, Boston, MA, USA.
Bradley T HymanDepartments of Neurology and Radiology, Massachusetts General Hospital and Alzheimer's Disease Research Center, Harvard Medical School, Boston, MA, USA.
Xandra O BreakefieldDepartments of Neurology and Radiology, Massachusetts General Hospital and Center for NeuroDiscovery, Harvard Medical School, Boston, MA, USA.
Martin IngelssonDepartments of Neurology and Radiology, Massachusetts General Hospital and Center for NeuroDiscovery, Harvard Medical School, Boston, MA, USA; Department of Public Health and Caring Sciences, Geriatrics, Rudbeck Laboratory, Uppsala University, Uppsala, Sweden. Electronic address: martin.ingelsson@pubcare.uu.se.

Funding

Transfer of microRNA regulators from glioblastoma to brain microenvironment U19CA179563 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI BREAKEFIELD, XANDRA OWENS, CHAREST, ALAIN · 2013 to 2017
$8.6M
Orchestra: A high performance biomedical supercomputing collaborativeS10RR028832 · NCRR · HARVARD MEDICAL SCHOOL · PI DANUSER, GAUDENZ · 2010 to 2010
$3.7M
NCI NIH HHS U19 CA179563NCRR NIH HHS S10 RR028832
6 · The paper itself

Abstract

The APPswe (Swedish) mutation in the amyloid precursor protein (APP) gene causes dominantly inherited Alzheimer's disease (AD) as a result of increased β-secretase cleavage of the amyloid-β (Aβ) precursor protein. This leads to abnormally high Aβ levels, not only in brain but also in peripheral tissues of mutation carriers. Here, we selectively disrupted the human mutant APP

Indexed as

adeno-associated virusAlzheimer's diseaseamyloid precursor proteinamyloid-βCRISPRgenome editingSwedish mutation

Identifiers

PMID29858078
PMCPMC5992788

What OpenQuestion holds

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Registered trials

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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.