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Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sick...

Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sick...

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_1825439334

Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and [Beta]-thalassemia

About this item

Full title

Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and [Beta]-thalassemia

Publisher

Washington: National Academy of Sciences

Journal title

Proceedings of the National Academy of Sciences - PNAS, 2016-09, Vol.113 (38), p.10661

Language

English

Formats

Publication information

Publisher

Washington: National Academy of Sciences

More information

Scope and Contents

Contents

Hereditary persistence of fetal hemoglobin (HPFH) is a condition in some individuals who have a high level of fetal hemoglobin throughout life. Individuals with compound heterozygous β-thalassemia or sickle cell disease (SCD) and HPFH have milder clinical manifestations. Using RNA-guided clustered regularly interspaced short palindromic repeats-ass...

Alternative Titles

Full title

Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and [Beta]-thalassemia

Identifiers

Primary Identifiers

Record Identifier

TN_cdi_proquest_journals_1825439334

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_1825439334

Other Identifiers

ISSN

0027-8424

E-ISSN

1091-6490

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