Stem Cell Editing Repairs Severe Immunodeficiency
Stem Cell Editing Repairs Severe Immunodeficiency
Scientists hoping to treat immunodeficiencies using gene therapy have found a way to edit stem cells in mice without disrupting gene regulation.
Scientists hoping to treat immunodeficiencies using gene therapy have found a way to edit stem cells in mice without disrupting gene regulation.
severe combined immunodeficiency syndrome (SCID)
Gene Therapy Continues to Benefit Kids with Immunodeficiency
Jef Akst | May 12, 2021 | 2 min read
Four dozen children with severe combined immunodeficiency (SCID) who received a corrective gene carried by a virus have working immune systems two to three years later, according to three independent clinical trials.