WISKOTT-ALDRICH SYNDROME GENE HOMING ENDONUCLEASE VARIANTS, COMPOSITIONS, AND METHODS OF USE
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United States of America Patent
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Issued Date -
Nov 17, 2022
app pub date -
Apr 24, 2020
filing date -
Apr 24, 2019
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Abstract
The present disclosure provides improved genome editing compositions and methods for editing a human Wiskott-Aldrich syndrome gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of WAS, including but not limited to, an immune system disorder, thrombocytopenia, eczema, X-linked thrombocytopenia (XLT), or X-linked neutropenia (XLN).
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