ReviewChromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology2015
Human artificial chromosomes for Duchenne muscular dystrophy and beyond: challenges and hopes.
Review in Chromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 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.
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Who cites it
12 citing papers in PubMed.
- Review
- Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin gene.Molecular therapy. Nucleic acids · 2023Article
- Engineering of human induced pluripotent stem cells via human artificial chromosome vectors for cell therapy and disease modeling.Molecular therapy. Nucleic acids · 2021Article
- Pluripotent stem cell-based gene therapy approach: human de novo synthesized chromosomes.Cellular and molecular life sciences : CMLS · 2021Review
- Review
- Human induced pluripotent stem cell models for the study and treatment of Duchenne and Becker muscular dystrophies.Therapeutic advances in neurological disorders · 2019Review
- Autologous Cell Therapy Approach for Duchenne Muscular Dystrophy using PiggyBac Transposons and Mesoangioblasts.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy.EMBO molecular medicine · 2018Article
- Method to Assemble Genomic DNA Fragments or Genes on Human Artificial Chromosome with Regulated Kinetochore Using a Multi-Integrase System.ACS synthetic biology · 2018Article
- piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.Nucleic acids research · 2016Article
- Stem Cell Differentiation Toward the Myogenic Lineage for Muscle Tissue Regeneration: A Focus on Muscular Dystrophy.Stem cell reviews and reports · 2015Review
- Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells.Nature protocols · 2015Article
Corrections and comments
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Authors and funding
1 author.
Funding
Abstract
Safe and efficacious vectors able to carry large or several transgenes are of key importance for gene therapy. Human artificial chromosomes can fulfil this essential requirement; moreover, they do not integrate into the host genome. However, drawbacks such as the low efficiency of chromosome transfer and their relatively complex engineering still limit their widespread use. In this article, I summarise the key steps that brought human artificial chromosomes into preclinical research for Duchenne muscular dystrophy, an X-linked, monogenic disorder. I will also review possible future pre-clinical and clinical perspectives for this technology.
Indexed as
Identifiers
25596829What OpenQuestion holds
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.