Adenosine deaminase (ADA) deficiency results in severe combined immunodeficiency, the first genetic disorder treated by gene therapy. Two different retroviral vectors were used to transfer ex vivo the human ADA minigene into bone marrow cells and peripheral blood lymphocytes from two patients undergoing exogenous enzyme replacement therapy. After 2 years of treatment, long-term survival of T and B lymphocytes, marrow cells, and granulocytes expressing the transferred ADA gene was demonstrated and resulted in normalization of the immune repertoire and restoration of cellular and humoral immunity. After discontinuation of treatment, T lymphocytes, derived from transduced peripheral blood lymphocytes, were progressively replaced by marrow-deri...
Background: We investigated the long-term outcome of gene therapy for severe combined immunodeficien...
In the last decade, gene therapy for adenosine deaminase deficiency has been developed as a successf...
Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare an...
Adenosine deaminase (ADA) deficiency results in severe combined immunodeficiency, the first genetic ...
Peripheral blood lymphocytes obtained from a patient affected by adenosine deaminase (ADA) deficienc...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency disease (SCID). ...
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase ...
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase ...
Peripheral blood lymphocytes obtained from a patient af-fected by adenosine deaminase (ADA) deficien...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency (SCID), a candid...
Adenosine deaminase (ADA) deficiency results in severe combined immune deficiency disease (SCID), wh...
Patients lacking functional adenosine deaminase activity have severe combined immunodeficiency (ADA ...
Adenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction, leading to severe ...
Genetic defects in the purine salvage enzyme adenosine deaminase (ADA) lead to severe combined immun...
Adenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction, leading to severe ...
Background: We investigated the long-term outcome of gene therapy for severe combined immunodeficien...
In the last decade, gene therapy for adenosine deaminase deficiency has been developed as a successf...
Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare an...
Adenosine deaminase (ADA) deficiency results in severe combined immunodeficiency, the first genetic ...
Peripheral blood lymphocytes obtained from a patient affected by adenosine deaminase (ADA) deficienc...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency disease (SCID). ...
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase ...
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase ...
Peripheral blood lymphocytes obtained from a patient af-fected by adenosine deaminase (ADA) deficien...
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency (SCID), a candid...
Adenosine deaminase (ADA) deficiency results in severe combined immune deficiency disease (SCID), wh...
Patients lacking functional adenosine deaminase activity have severe combined immunodeficiency (ADA ...
Adenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction, leading to severe ...
Genetic defects in the purine salvage enzyme adenosine deaminase (ADA) lead to severe combined immun...
Adenosine deaminase (ADA) deficiency is caused by a purine metabolic dysfunction, leading to severe ...
Background: We investigated the long-term outcome of gene therapy for severe combined immunodeficien...
In the last decade, gene therapy for adenosine deaminase deficiency has been developed as a successf...
Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare an...