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Optimized lentiviral vector for xla gene therapy

A carrier and polynucleotide technology, applied in gene therapy, medical raw materials derived from viruses/phages, viruses/phages, etc.

Pending Publication Date: 2020-02-18
SEATTLE CHILDRENS HOSPITAL (DBA SEATTLE CHILDRENS RES INST)
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Despite this therapy, XLA subjects continue to suffer from chronic infection and are at increased risk for a range of morbid or life-threatening complications

Method used

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  • Optimized lentiviral vector for xla gene therapy
  • Optimized lentiviral vector for xla gene therapy
  • Optimized lentiviral vector for xla gene therapy

Examples

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Embodiment Construction

[0147] BTK is expressed on both B cells and myeloid cells, where BTK also contributes to normal functional responses in both lineages. Failure to express BTK results in XLA. Conversely, overexpression of activated BTK or wild-type BTK leads to cellular transformation and / or developmental arrest ("Early arrest in B cell development in transgenic mice that express the E41KBruton's tyrosine kinase mutant under the control of the CD19 promoterregion" J Immunol .1999Jun 1; 162(11):6526-33; "Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized humanBTK." Leukemia.2010Sep; 24(9):1617-30; cited incorporated herein in its entirety), and dysregulated expression of wild-type BTK can promote autoantibody production and increase the risk of autoimmunity (“Enhanced Expression of Bruton's Tyrosine Kinase in B Cells Drives Systemic Autoimmunity by Disrupting T Cell Homeostasis.” J Immunol. 2016 Jul 1; 197(1):58-67; incorporated herein by refere...

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Abstract

Described herein are compositions and methods for treating, inhibiting or ameliorating X linked agammaglobulinemia (XLA) in subjects that have been identified or selected as being ones that would benefit from a therapy to treat, inhibit, or ameliorate XLA. Exemplary embodiments include constructs and methods for gene therapy, which restore or increase BTK expression.

Description

[0001] Incorporate any priority application by reference into [0002] This application claims the benefit of priority to U.S. Provisional Patent Application No. 62 / 488,523, filed April 21, 2017. The entire disclosure of the above application is expressly incorporated by reference in its entirety. [0003] References to Federal Funds [0004] This invention was made with support under Grant No. AI084457 awarded by the NIH National Institute of Allergy and Infectious Diseases. [0005] References to Sequence Listings [0006] Submit this application together with the sequence listing in electronic format. The Sequence Listing is provided as a file titled Sequence ListingSCRI.148WO.txt, created on April 17, 2018, and is 140kb in size. The information in the sequence listing in electronic format is hereby incorporated by reference in its entirety. technical field [0007] Aspects of the present invention relate to compositions and methods for treating, inhibiting or alleviat...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/54C12N15/85C12N5/10A61K48/00A61K35/76A61K35/28A61P37/04
CPCA61K35/28C12N15/86A61P37/04A61K38/00A61K48/0058C12N2740/16043C12N2800/22C12N2830/008C12N2830/46C12N9/1205A61K35/17C12N7/00C12N9/12C12N2740/15043C12Y207/10002
Inventor 戴维德·J·罗林斯凯伦·索默斯瓦蒂·辛格
Owner SEATTLE CHILDRENS HOSPITAL (DBA SEATTLE CHILDRENS RES INST)
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