ReviewWorld journal of stem cells2016
Human induced pluripotent stem cells for monogenic disease modelling and therapy.
Review in World journal of stem cells, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
What it found
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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.
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.
Who cites it
18 citing papers in PubMed.
- Stem cell-based therapies in pediatric disorders: translational advances, unresolved challenges, and future horizons.Stem cell research & therapy · 2026Review
- Early considerations for village-in-a-dish platform: a multidisciplinary perspective.Frontiers in bioengineering and biotechnology · 2026Review
- Integrating population genetics, stem cell biology and cellular genomics to study complex human diseases.Nature genetics · 2024Article
- Advances in the Treatment of Kidney Disorders using Mesenchymal Stem Cells.Current pharmaceutical design · 2024Review
- Myc beyond Cancer: Regulation of Mammalian Tissue Regeneration.Pathophysiology : the official journal of the International Society for Pathophysiology · 2023Review
- Development of brain organoid technology derived from iPSC for the neurodegenerative disease modelling: a glance through.Frontiers in molecular neuroscience · 2023Review
- Development of vascular disease models to explore disease causation and pathomechanisms of rare vascular diseases.Seminars in immunopathology · 2022Review
- Human pluripotent stem cell-derived models and drug screening in CNS precision medicine.Annals of the New York Academy of Sciences · 2020Review
- Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery.Human genomics · 2020Review
- Functional diversity of small nucleolar RNAs.Nucleic acids research · 2020Article
- Generation and Neuronal Differentiation of hiPSCs From Patients With Myotonic Dystrophy Type 2.Frontiers in physiology · 2018Article
- A pathologist's perspective on induced pluripotent stem cells.Laboratory investigation; a journal of technical methods and pathology · 2017Review
- Exposure of Induced Pluripotent Stem Cell-Derived Vascular Endothelial and Smooth Muscle Cells in Coculture to Hemodynamics Induces Primary Vascular Cell-Like Phenotypes.Stem cells translational medicine · 2017Article
- What Can be Learned from the Time Course of Changes in Low-Frequency Stimulated Muscle?European journal of translational myology · 2017Article
- A preliminary analysis of volatile metabolites of human induced pluripotent stem cells along the in vitro differentiation.Scientific reports · 2017Article
- Generation of induced Pluripotent Stem Cells as disease modelling of NLSDM.Molecular genetics and metabolism · 2017Article
- Regenerative Medicine: lessons from Mother Nature.Regenerative medicine · 2016Article
- Cell Therapy of Corneal Diseases.Cornea · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Recent and advanced protocols are now available to derive human induced pluripotent stem cells (hiPSCs) from patients affected by genetic diseases. No curative treatments are available for many of these diseases; thus, hiPSCs represent a major impact on patient' health. hiPSCs represent a valid model for the in vitro study of monogenic diseases, together with a better comprehension of the pathogenic mechanisms of the pathology, for both cell and gene therapy protocol applications. Moreover, these pluripotent cells represent a good opportunity to test innovative pharmacological treatments focused on evaluating the efficacy and toxicity of novel drugs. Today, innovative gene therapy protocols, especially gene editing-based, are being developed, allowing the use of these cells not only as in vitro disease models but also as an unlimited source of cells useful for tissue regeneration and regenerative medicine, eluding ethical and immune rejection problems. In this review, we will provide an up-to-date of modelling monogenic disease by using hiPSCs and the ultimate applications of these in vitro models for cell therapy. We consider and summarize some peculiar aspects such as the type of parental cells used for reprogramming, the methods currently used to induce the transcription of the reprogramming factors, and the type of iPSC-derived differentiated cells, relating them to the genetic basis of diseases and to their inheritance model.
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Registered trials
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.