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Design Principles of a Novel Construct for HBB Gene-Editing and Investigation of Its Gene-Targeting...

Design Principles of a Novel Construct for HBB Gene-Editing and Investigation of Its Gene-Targeting...

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_3153600086

Design Principles of a Novel Construct for HBB Gene-Editing and Investigation of Its Gene-Targeting Efficiency in HEK293 Cells

About this item

Full title

Design Principles of a Novel Construct for HBB Gene-Editing and Investigation of Its Gene-Targeting Efficiency in HEK293 Cells

Publisher

New York: Springer US

Journal title

Molecular biotechnology, 2024-03, Vol.66 (3), p.517-530

Language

English

Formats

Publication information

Publisher

New York: Springer US

More information

Scope and Contents

Contents

Beta-thalassemia is one of the most common monogenic inherited disorders worldwide caused by different mutations in the hemoglobin subunit beta (
HBB
) gene. Genome-editing based on clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 system (CRISPR/Cas9) has raised the hope for life-long gene therapy of beta-...

Alternative Titles

Full title

Design Principles of a Novel Construct for HBB Gene-Editing and Investigation of Its Gene-Targeting Efficiency in HEK293 Cells

Identifiers

Primary Identifiers

Record Identifier

TN_cdi_proquest_miscellaneous_3153600086

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_3153600086

Other Identifiers

ISSN

1073-6085

E-ISSN

1559-0305

DOI

10.1007/s12033-023-00739-6

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