Evidence map›Paper›PMID 31500113›Full record

ReviewInternational journal of molecular sciences2019

Gene Therapy for ALS-A Perspective.

Marisa Cappella, Chiara Ciotti, Mathilde Cohen-Tannoudji, Maria Grazia Biferi

Open access · goldAbstract readReview
In one paragraph

Review in International journal of molecular sciences, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 51 papers.

0numbers the graph read from it
0cells of the map it votes in
51citing papers in PubMed
9.7field-weighted citation impact, top 1% 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

51 citing papers in PubMed, 96 citations in OpenAlex.

  1. Review
  2. Review
  3. Review
  4. Article
  5. Review
  6. Targets and Gene Therapy of ALS (Part 1).International journal of molecular sciences · 2025
    Review
  7. Review
  8. Review
  9. Energy metabolism in health and diseases.Signal transduction and targeted therapy · 2025
    Review
  10. Mechanisms underlining R-loop biology and implications for human disease.Frontiers in cell and developmental biology · 2025
    Review
  11. Review
  12. Skeletal muscle dysfunction in amyotrophic lateral sclerosis: a mitochondrial perspective and therapeutic approaches.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024
    Review
  13. Article
  14. In Vitro Models of Amyotrophic Lateral Sclerosis.Cellular and molecular neurobiology · 2023
    Review
  15. Review
  16. Gene Therapy in ALS and SMA: Advances, Challenges and Perspectives.International journal of molecular sciences · 2023
    Review
  17. Article
  18. Article
  19. Article
  20. Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges.Molecular therapy : the journal of the American Society of Gene Therapy · 2022
    Review
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

4 authors at 1 institution in 1 country.

Marisa CappellaSorbonne Université, Inserm UMRS 974, Centre of Research in Myology (CRM), Institut de Myologie, GH Pitié Salpêtrière, 75013 Paris, France.
Chiara CiottiSorbonne Université, Inserm UMRS 974, Centre of Research in Myology (CRM), Institut de Myologie, GH Pitié Salpêtrière, 75013 Paris, France.
Mathilde Cohen-TannoudjiSorbonne Université, Inserm UMRS 974, Centre of Research in Myology (CRM), Institut de Myologie, GH Pitié Salpêtrière, 75013 Paris, France.
Maria Grazia BiferiSorbonne Université, Inserm UMRS 974, Centre of Research in Myology (CRM), Institut de Myologie, GH Pitié Salpêtrière, 75013 Paris, France. mg.biferi@institut-myologie.org.
Inserm · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease (MND) with no cure. Recent advances in gene therapy open a new perspective to treat this disorder-particularly for the characterized genetic forms. Gene therapy approaches, involving the delivery of antisense oligonucleotides into the central nervous system (CNS) are being tested in clinical trials for patients with mutations in

Indexed as

Genetic TherapyAmyotrophic Lateral SclerosisAnimalsC9orf72 ProteinDisease Models, AnimalGene EditingGene ExpressionGenetic Predisposition to DiseaseGenetic VectorsGene Transfer TechniquesHumansMolecular Targeted TherapyMotor NeuronsMutationSuperoxide Dismutase-1TransgenesC9orf72 ProteinC9orf72 protein, humanSuperoxide Dismutase-1AAVantisense oligonucleotidesGene therapylentiviral vectorsRNA interference

Identifiers

PMID31500113
PMCPMC6771059
OpenAlexW2972177292

What Socratic holds

Textmetadata
LicenceCC BY
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 Socratic graph.