T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency.

ABSTRACT:

Background

Mutation of the IL2RG gene results in a form of severe combined immune deficiency (SCID-X1), which has been treated successfully with hematopoietic stem cell gene therapy. SCID-X1 gene therapy results in reconstitution of the previously lacking T cell compartment, allowing analysis of the roles of T cell immunity in humans by comparing before and after gene correction.

Methods

Here we interrogate T cell reconstitution using four forms of high throughput analysis. (1) Estimation of the numbers of transduced progenitor cells by monitoring unique positions of integration of the therapeutic gene transfer vector. (2) Estimation of T cell population structure by sequencing of the recombined T cell receptor (TCR) beta locus. (3) Metagenomic analysis of microbial populati

SUBMITTER: Clarke EL 

PROVIDER: S-EPMC6161392 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature