Add to ORKGIn 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA- SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease
Genetic defects in the adenosine deaminase (ADA) gene are among the most common causes for severe c...
Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare an...
Gene transfer into hematopoietic stem cells by γ-retroviral vectors (RVs) is an effective treatment ...
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase ...
Adenosine deaminase (ADA) deficiency results in severe combined immunodeficiency, the first genetic ...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency (SCID), a candid...
Peripheral blood lymphocytes obtained from a patient affected by adenosine deaminase (ADA) deficienc...
Peripheral blood lymphocytes obtained from a patient af-fected by adenosine deaminase (ADA) deficien...
Patients lacking functional adenosine deaminase activity have severe combined immunodeficiency (ADA ...
Claudia A Montiel-Equihua, Adrian J Thrasher, H Bobby GasparCentre for Immunodeficiency, Molecular I...
Gene therapy is a highly attractive strategy for many types of inherited disorders of the immune sys...
BACKGROUND: We investigated the long-term outcome of gene therapy for severe combined immunodeficien...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency disease (SCID). ...
The use of gene therapy in the treatment of primary immune deficiencies (PID) has advanced significa...
Adenosine deaminase (ADA) deficiency is a rare, autosomal-recessive systemic metabolic disease chara...
Genetic defects in the adenosine deaminase (ADA) gene are among the most common causes for severe c...
Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare an...
Gene transfer into hematopoietic stem cells by γ-retroviral vectors (RVs) is an effective treatment ...
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase ...
Adenosine deaminase (ADA) deficiency results in severe combined immunodeficiency, the first genetic ...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency (SCID), a candid...
Peripheral blood lymphocytes obtained from a patient affected by adenosine deaminase (ADA) deficienc...
Peripheral blood lymphocytes obtained from a patient af-fected by adenosine deaminase (ADA) deficien...
Patients lacking functional adenosine deaminase activity have severe combined immunodeficiency (ADA ...
Claudia A Montiel-Equihua, Adrian J Thrasher, H Bobby GasparCentre for Immunodeficiency, Molecular I...
Gene therapy is a highly attractive strategy for many types of inherited disorders of the immune sys...
BACKGROUND: We investigated the long-term outcome of gene therapy for severe combined immunodeficien...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency disease (SCID). ...
The use of gene therapy in the treatment of primary immune deficiencies (PID) has advanced significa...
Adenosine deaminase (ADA) deficiency is a rare, autosomal-recessive systemic metabolic disease chara...
Genetic defects in the adenosine deaminase (ADA) gene are among the most common causes for severe c...
Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare an...
Gene transfer into hematopoietic stem cells by γ-retroviral vectors (RVs) is an effective treatment ...