Gene transfer into autologous hematopoietic stem cells by γ-retroviral vectors (gRV) is an effective treatment for adenosine deaminase (ADA)–deficient severe combined immunodeficiency (SCID). However, current gRV have significant potential for insertional mutagenesis as reported in clinical trials for other primary immunodeficiencies. To improve the efficacy and safety of ADA-SCID gene therapy (GT), we generated a self-inactivating lentiviral vector (LV) with a codon-optimized human cADA gene under the control of the short form elongation factor-1α promoter (LV EFS ADA). In ADA−/− mice, LV EFS ADA displayed high-efficiency gene transfer and sufficient ADA expression to rescue ADA−/− mice from their lethal phenotype with good thymic and peri...
Patients lacking functional adenosine deaminase activity have severe combined immunodeficiency (ADA ...
X-linked severe combined immunodeficiency (SCID-X1) is caused by mutations in the interleukin-2 rece...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency disease (SCID). ...
Claudia A Montiel-Equihua, Adrian J Thrasher, H Bobby GasparCentre for Immunodeficiency, Molecular I...
Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare an...
Mutations in the Adenosine Deaminase (ADA) gene are responsible for a form of Severe Combined Immuno...
Adenosine deaminase (ADA) deficiency is a rare, autosomal-recessive systemic metabolic disease chara...
Background: We investigated the long-term outcome of gene therapy for severe combined immunodeficien...
UnrestrictedI have investigated three different therapeutic approaches for the treatment of adenosin...
Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative t...
Adenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction, leading to severe ...
Adenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction, leading to severe ...
Deficiency of adenosine deaminase type 2 (DADA2) is an autosomal recessive disease caused by bi-alle...
Peripheral blood lymphocytes obtained from a patient affected by adenosine deaminase (ADA) deficienc...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency (SCID), a candid...
Patients lacking functional adenosine deaminase activity have severe combined immunodeficiency (ADA ...
X-linked severe combined immunodeficiency (SCID-X1) is caused by mutations in the interleukin-2 rece...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency disease (SCID). ...
Claudia A Montiel-Equihua, Adrian J Thrasher, H Bobby GasparCentre for Immunodeficiency, Molecular I...
Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare an...
Mutations in the Adenosine Deaminase (ADA) gene are responsible for a form of Severe Combined Immuno...
Adenosine deaminase (ADA) deficiency is a rare, autosomal-recessive systemic metabolic disease chara...
Background: We investigated the long-term outcome of gene therapy for severe combined immunodeficien...
UnrestrictedI have investigated three different therapeutic approaches for the treatment of adenosin...
Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative t...
Adenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction, leading to severe ...
Adenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction, leading to severe ...
Deficiency of adenosine deaminase type 2 (DADA2) is an autosomal recessive disease caused by bi-alle...
Peripheral blood lymphocytes obtained from a patient affected by adenosine deaminase (ADA) deficienc...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency (SCID), a candid...
Patients lacking functional adenosine deaminase activity have severe combined immunodeficiency (ADA ...
X-linked severe combined immunodeficiency (SCID-X1) is caused by mutations in the interleukin-2 rece...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency disease (SCID). ...