Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies
Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies
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Publisher
Switzerland: MDPI
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Language
English
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Publisher
Switzerland: MDPI
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Contents
Background and objectives: Choroideremia (CHM) is an X-linked recessive chorioretinal dystrophy caused by mutations involving the CHM gene. Gene therapy has entered late-phase clinical trials, although there have been variable results. This review gives a summary on the outcomes of phase I/II CHM gene therapy trials and describes other potential ex...
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Full title
Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies
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TN_cdi_doaj_primary_oai_doaj_org_article_5fe63729fc2f4758a15480575ed287c8
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_5fe63729fc2f4758a15480575ed287c8
Other Identifiers
ISSN
1648-9144,1010-660X
E-ISSN
1648-9144
DOI
10.3390/medicina57010064
