Generation of X-CGD cells for vector evaluation from healthy donor CD34+ HSCs by shRNA-mediated knoc...
Generation of X-CGD cells for vector evaluation from healthy donor CD34+ HSCs by shRNA-mediated knock down of gp91phox
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Publisher
Milwaukee: Elsevier Limited
Journal title
Language
English
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Publisher
Milwaukee: Elsevier Limited
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Scope and Contents
Contents
Innovative approaches for the treatment of rare inherited diseases are hampered by limited availability of patient derived samples for preclinical research. This also applies for the evaluation of novel vector systems for the gene therapy of monogenic hematological diseases like X-linked chronic granulomatous disease (X-CGD), a severe primary immun...
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Full title
Generation of X-CGD cells for vector evaluation from healthy donor CD34+ HSCs by shRNA-mediated knock down of gp91phox
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TN_cdi_doaj_primary_oai_doaj_org_article_833b07f507eb4f928a8ffe1fb7660c4e
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_833b07f507eb4f928a8ffe1fb7660c4e
Other Identifiers
ISSN
2329-0501
E-ISSN
2329-0501
DOI
10.1038/mtm.2014.37
