New Gene Therapy Potential Cure for "Bubble Boy Disease": An experimental gene therapy has allowed children with SCID-1X to develop fully functioning immune systems.

Subjects

Subjects :

Busulfan therapeutic use
Child
Child, Preschool
Female
Gene Expression
Genetic Vectors chemistry
Genetic Vectors metabolism
Humans
Immune System drug effects
Immune System immunology
Immune System pathology
Immunosuppressive Agents therapeutic use
Infant
Interleukin Receptor Common gamma Subunit deficiency
Interleukin Receptor Common gamma Subunit immunology
Lentivirus metabolism
Male
T-Lymphocytes drug effects
T-Lymphocytes immunology
T-Lymphocytes pathology
X-Linked Combined Immunodeficiency Diseases genetics
X-Linked Combined Immunodeficiency Diseases immunology
X-Linked Combined Immunodeficiency Diseases pathology
Genetic Therapy methods
Interleukin Receptor Common gamma Subunit genetics
Lentivirus genetics
Therapies, Investigational methods
X-Linked Combined Immunodeficiency Diseases therapy

Details

Language :

English

ISSN :

1552-4833

Volume :

179

Issue :

7

Database :

MEDLINE

Journal :

American journal of medical genetics. Part A

Publication Type :

Academic Journal

Accession number :

31180192

Full Text :

https://doi.org/10.1002/ajmg.a.40453