Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia

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.

Description

[0001] Cross References to Related Applications [0002] This application claims the benefit of priority to U.S. Provisional Patent Application No. 62 / 488,249, filed April 21, 2017. The entire disclosure of the above application is expressly incorporated by reference in its entirety. [0003] References to Sequence Listings [0004] Submit this application together with the sequence listing in electronic format. The Sequence Listing is provided as a file titled 2018-04-19 Sequence Listing-SCRI.149WO.txt created on April 19, which is 158kb in size. The information in the sequence listing in electronic format is expressly incorporated by reference in its entirety. technical field [0005] The alternative approach herein generally involves gene editing in primary human hematopoietic stem cells. More specifically, the alternatives herein relate to nucleic acids and vectors configured to provide efficient homology-mediated gene repair, and methods of repairing genetic defects s...

AI Technical Summary

Problems solved by technology

Viral-based gene replacement is also being explored in several current clinical trials, but more therapies for WAS and XLT are clearly needed

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