Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders.
chromosome transfer
chromosome transplantation
genetic therapy
genomic disorders
induced pluripotent stem cells
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
12 Jun 2020
12 Jun 2020
Historique:
received:
21
10
2019
accepted:
07
01
2020
entrez:
27
2
2020
pubmed:
27
2
2020
medline:
27
2
2020
Statut:
epublish
Résumé
Many human genetic diseases are associated with gross mutations such as aneuploidies, deletions, duplications, or inversions. For these "structural" disorders, conventional gene therapy, based on viral vectors and/or on programmable nuclease-mediated homologous recombination, is still unsatisfactory. To correct such disorders, chromosome transplantation (CT), defined as the perfect substitution of an endogenous defective chromosome with an exogenous normal one, could be applied. CT re-establishes a normal diploid cell, leaving no marker of the procedure, as we have recently shown in mouse pluripotent stem cells. To prove the feasibility of the CT approach in human cells, we used human induced pluripotent stem cells (hiPSCs) reprogrammed from Lesch-Nyhan (LN) disease patients, taking advantage of their mutation in the X-linked
Identifiants
pubmed: 32099849
doi: 10.1016/j.omtm.2020.01.003
pii: S2329-0501(20)30014-0
pmc: PMC7029378
doi:
Types de publication
Journal Article
Langues
eng
Pagination
369-377Informations de copyright
© 2020 The Author(s).
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