Evidence map›Paper›PMID 28616376›Full record

ReviewCurrent stem cell reports2017

Genome Editing and Muscle Stem Cells as a Therapeutic Tool for Muscular Dystrophies.

Veronica Pini, Jennifer E Morgan, Francesco Muntoni, Helen C O'Neill

Open access · hybridAbstract readReview
In one paragraph

Review in Current stem cell reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
1.7field-weighted citation impact, top 16% 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

14 citing papers in PubMed, 18 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Article
  5. Review
  6. Review
  7. Article
  8. Article
  9. Review
  10. Pericytes in Muscular Dystrophies.Advances in experimental medicine and biology · 2019
    Article
  11. Article
  12. Review
  13. Review
  14. 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

4 authors at 2 institutions in 2 countries.

Veronica PiniMolecular and Developmental Neurosciences Program, The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH UK.
Jennifer E MorganMolecular and Developmental Neurosciences Program, The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH UK.
Francesco MuntoniMolecular and Developmental Neurosciences Program, The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London, WC1N 1EH UK.
Helen C O'NeillEmbryology, IVF and Reproductive Genetics Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London, WC1E 6HX UK.
University College London · GBInstitute for Reproductive Health · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purpose of reviewMuscular dystrophies are a group of severe degenerative disorders characterized by muscle fiber degeneration and death. Therapies designed to restore muscle homeostasis and to replace dying fibers are being experimented, but none of those in clinical trials are suitable to permanently address individual gene mutation. The purpose of this review is to discuss genome editing tools such as CRISPR/Cas (clustered regularly interspaced short palindromic repeats/CRISPR-associated), which enable direct sequence alteration and could potentially be adopted to correct the genetic defect leading to muscle impairment. RECENT

findingsRecent findings show that advances in gene therapy, when combined with traditional viral vector-based approaches, are bringing the field of regenerative medicine closer to precision-based medicine. SUMMARY: The use of such programmable nucleases is proving beneficial for the creation of more accurate in vitro and in vivo disease models. Several gene and cell-therapy studies have been performed on satellite cells, the primary skeletal muscle stem cells involved in muscle regeneration. However, these have mainly been based on artificial replacement or augmentation of the missing protein. Satellite cells are a particularly appealing target to address these innovative technologies for the treatment of muscular dystrophies.

Indexed as

CRISPR/CasGene therapyGenome editingMuscular dystrophiesPrecision medicineStem cells

Identifiers

PMID28616376
PMCPMC5445179
OpenAlexW2607005583

What OpenQuestion 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 OpenQuestion graph.