Related Experiment Video
Updated: Jul 8, 2026

A Syngeneic Mouse B-Cell Lymphoma Model for Pre-Clinical Evaluation of CD19 CAR T Cells
Published on: October 16, 2018
Gene therapy for immune disorders: good news tempered by bad news
Jennifer M Puck1, Harry L Malech
1Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA. jpuck@mail.nih.gov
Abstract:
After a dozen years of human gene therapy trials characterized by minimal gene correction and disappointing clinical impact, the field of gene therapy received some good news in 2000. Infants with X-linked severe combined immunodeficiency who received retroviral gene addition to cells from their bone marrow developed impressive immune reconstitution. During the following 2 years, additional patients were treated and the news was even better-babies receiving gene therapy had sustained T-cell production and in several cases developed better cell function than most patients treated with standard bone marrow transplants. Unfortunately, bad news followed. Three of the patients experienced leukemic T-cell expansions, found to be associated with retroviral insertions into genomic DNA. Where does the field stand today?
Related Concept Videos
What is Genetic Engineering?
Gene Therapy
Gene Therapy
Autoimmune Disorders
Concept and Mechanism of Autoimmune Diseases
The immune system...
Tumor Immunotherapy
Microorganisms in Medicine and Therapeutics

