Normal IgH Repertoire Diversity in an Infant with ADA Deficiency After Gene Therapy.

ABSTRACT:

Purpose

Adenosine deaminase (ADA) deficiency causes severe combined immunodeficiency (SCID) through an accumulation of toxic metabolites within lymphocytes. Recently, ADA deficiency has been successfully treated using lentiviral-transduced autologous CD34+ cells carrying the ADA gene. T and B cell function appears to be fully restored, but in many patients' B cell numbers remain low, and assessments of the immunoglobulin heavy (IgHV) repertoire following gene therapy are lacking.

Methods

We performed deep sequencing of IgHV repertoire in peripheral blood lymphocytes from a child following lentivirus-based gene therapy for ADA deficiency and compared to the IgHV repertoire in healthy infants and adults.

Results

After gene therapy, Ig diversity increased over time as ev

SUBMITTER: Baloh CH 

PROVIDER: S-EPMC9906566 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature