Therapy Development by Genome Editing of Hematopoietic Stem Cells.
Cells
; 10(6)2021 06 14.
Article
em En
| MEDLINE
| ID: mdl-34198536
Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the blood and immune systems has placed them at the forefront of cell and gene therapy development. Recent advances in genome-editing tools, in particular for clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) and CRISPR/Cas-derived editing systems, have transformed the gene therapy landscape. Their versatility and the ability to edit genomic sequences and facilitate gene disruption, correction or insertion, have broadened the spectrum of potential gene therapy targets and accelerated the development of potential curative therapies for many rare diseases treatable by transplantation or modification of HSCs. Ongoing developments seek to address efficiency and precision of HSC modification, tolerability of treatment and the distribution and affordability of corresponding therapies. Here, we give an overview of recent progress in the field of HSC genome editing as treatment for inherited disorders and summarize the most significant findings from corresponding preclinical and clinical studies. With emphasis on HSC-based therapies, we also discuss technical hurdles that need to be overcome en route to clinical translation of genome editing and indicate advances that may facilitate routine application beyond the most common disorders.
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Base de dados:
MEDLINE
Assunto principal:
Células-Tronco Hematopoéticas
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Terapia Genética
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Sistemas CRISPR-Cas
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Edição de Genes
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Doenças Genéticas Inatas
Idioma:
En
Ano de publicação:
2021
Tipo de documento:
Article