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This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

Innovation Observatory > Reports > Drugs > Autologous CD34+ hematopoietic stem cells transduced ex vivo with lentiviral vector encoding for the human ADA gene for severe combined immunodeficiency caused by adenosine deaminase deficiency

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Autologous CD34+ hematopoietic stem cells transduced ex vivo with lentiviral vector encoding for the human ADA gene for severe combined immunodeficiency caused by adenosine deaminase deficiency

Drugs

Endocrine, Nutritional and Metabolic

September 2016


Ex vivo lentiviral gene therapy is a new treatment for a type of severe immunodeficiency caused by lack of a specific enzyme, meaning the patient’s body is unable to fight infections. Current patients require stem cell transplants and regular injections of a replacement enzyme. Ex vivo lentiviral gene therapy modifies the patient’s cells to create the missing enzyme to potentially cure the condition.

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