Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia

Described herein are systems and methods for treating, inhibiting, or ameliorating X-linked disorders including Wiskott-Aldrich Syndrome (WAS) and X-linked thrombocytopenia (XLT) in subjects that have been identified or selected as being ones that would benefit from a therapy to treat, inhibit, or a...

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Published 09.05.2023
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Abstract Described herein are systems and methods for treating, inhibiting, or ameliorating X-linked disorders including Wiskott-Aldrich Syndrome (WAS) and X-linked thrombocytopenia (XLT) in subjects that have been identified or selected as being ones that would benefit from a therapy to treat, inhibit, or ameliorate WAS or XLT. The systems include nuclease and vector donor constructs configured for co-delivery to modify endogenous WAS locus.
AbstractList Described herein are systems and methods for treating, inhibiting, or ameliorating X-linked disorders including Wiskott-Aldrich Syndrome (WAS) and X-linked thrombocytopenia (XLT) in subjects that have been identified or selected as being ones that would benefit from a therapy to treat, inhibit, or ameliorate WAS or XLT. The systems include nuclease and vector donor constructs configured for co-delivery to modify endogenous WAS locus.
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References (WO 19/209912) 20191000
Ohlmann (10968253) 20210400
(WO-2018058064) 20180300
(WO 2016/057961) 20160400
(WO 19/210216) 20191000
(WO 15/057980) 20150400
(WO 15/048577) 20150400
Zhang (20060134673) 20060600
Then et al. (20200006322) 20200700
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Snippet Described herein are systems and methods for treating, inhibiting, or ameliorating X-linked disorders including Wiskott-Aldrich Syndrome (WAS) and X-linked...
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Title Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia
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