Hematopoietic stem cell gene therapy (GT) using a γ-retroviral vector (γ-RV) is an effective
treatment for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency …

Two patients with adenosine deaminase (ADA)-deficient severe combined
immunodeficiency (ADA-SCID) received stem cell-based gene therapy (SCGT) using …

Gene transfer into hematopoietic stem cells by γ-retroviral vectors (RVs) is an effective
treatment for inherited blood disorders, although potentially limited by the risk of insertional …

For X-linked severe combined immunodeficiency (SCID-X1), the practice of gene therapy
has revealed an unusual effect: insertional mutagenesis that can lead to leukemia. Even …

Retroviral gene therapy has proved efficacious for multiple genetic diseases of the
hematopoietic system, but roughly half of clinical gene therapy trial protocols using …

The Committee for Human Medicinal Products has recently recommended marketing
authorization for retrovirus-mediated ex vivo gene therapy of patients' autologous CD34+ …

Severe combined immunodeficiency (SCID) caused by adenosine deaminase deficiency
(ADA–) is the first genetic disorder to be treated with gene therapy. Since 1990 when the first …

Retroviruses were used as the basis for the first eukaryotic viral vectors and were the first
viral vectors to be employed in human clinical trials [1]. Since then, retroviral vectors have …

GlaxoSmithKline's (GSK) and partner San Raffaele Telethon Institute for Gene Therapy's
recent positive European approval for Strimvelis for treatment of severe combined …

Biallelic defects in the ADA gene cause deficiency of ADA, the enzyme that catalyzes the
conversion of adenosine to inosine. Lack of ADA activity results in accumulation of …