Inborn errors of immunity (IEI) are diseases caused by genetic mutations that affect the
immune system's ability to fight pathogens, cope with the microbiota or regulate …

Adenosine deaminase (ADA) is an enzyme of the purine metabolism which catalyzes the
irreversible deamination of adenosine and deoxyadenosine to inosine and deoxyinosine …

Hematopoietic stem cell (HSC) gene therapy for adenosine deaminase (ADA)–deficient
severe combined immunodeficiency (SCID) has shown limited clinical efficacy because of …

Genetic defects in the purine salvage enzyme adenosine deaminase (ADA) lead to severe
combined immunodeficiency (SCID) with profound depletion of T, B, and natural killer cell …

Ex vivo retrovirally mediated gene therapy has been shown within the last 20 yr to correct
the T cell immunodeficiency caused by γc-deficiency (SCID X1) and adenosine deaminase …

Abstract Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic
diseases caused by functional defects in specific lysosomal proteins. The lysosome is a …

A key factor in the success of gene therapy is the development of gene delivery systems that
are capable of efficient gene transfer in a broad variety of tissues, without causing any …

We compared the efficiency of transduction by an HIV-1-based lentiviral vector to that by a
Moloney murine leukemia virus (MLV) retroviral vector, using stringent in vitro assays of …

Twenty-five years have passed since first attempts of gene therapy (GT) in children affected
by severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) defect …

Adenosine deaminase-deficient severe combined immunodeficiency was the first disease
investigated for gene therapy because of a postulated production or survival advantage for …