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

A syndrome, gene technology, applied in the field of repairing genetic defects

Pending Publication Date: 2020-02-11
SEATTLE CHILDRENS HOSPITAL (DBA SEATTLE CHILDRENS RES INST)
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

Viral-based gene replacement is also being explored in several curr...

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

Examples

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Embodiment 2

[0142] Examples 2 and 3 show that, using the T7 endonuclease assay, frequencies of 85% and 73% were achieved with TALENs and CRISPR / Cas systems, respectively. These examples also show a method that elicits 70% homology-mediated repair (HDR) in T cells when nucleases are co-delivered with AAV donor templates.

[0143] Alternative 4: Editing of mobilized adult CD34+ cells with co-delivery of TALENs and AAV

[0144] This alternative demonstrates the effect of co-delivery of TALENs and AAVs on mobilized adult CD34 as described in some alternatives herein. + Methods for cell editing.

[0145] As described in Example 2 with TALEN and AAV against CD34 + Cells are transfected. Figure 4A A timeline of experimental conditions is shown where cells were analyzed by flow cytometry on days 2, 5 and 8. Figure 4B Shown are the GFP% on days 2 and 5, and the cell viability at Figure 4C is depicted in . Figure 4D Representative FACS plots showing GFP expression at day 5 are shown.

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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.

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...

Claims

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Application Information

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IPC IPC(8): A61K35/28A61K48/00A61P37/04
CPCA61K48/00A61K35/00C12N9/22C12N15/907C12N2750/14143A61P37/04
Inventor 戴维德·J·罗林斯伊拉姆·卡恩
Owner SEATTLE CHILDRENS HOSPITAL (DBA SEATTLE CHILDRENS RES INST)
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