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NEJM: Autologous lentiviral gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency

 Last Update: 2021-06-01
 Source: Internet
 Author: User

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Severe combined immunodeficiency (ADA-SCID) caused by adenosine deaminase (ADA) deficiency is a rare and life-threatening primary immunodeficiency.

Immune infection recently researchers examined the therapeutic effect of autologous in vitro lentiviral gene therapy for ADA-SCID is.

Fifty ADA-SCID patients (30 from the United States and 20 from the United Kingdom) participated in the study, using autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) to transduce a self-inactivating lentiviral vector encoding human ADA in vitro , The study was followed up for 24-36 months.

stem cell

During the follow-up, the overall survival rate of the patients was 100% .

The overall survival rate of patients was 100% .

Event-free survival rate of patients in the UK and US cohorts

Isolated lentiviral HSPC gene therapy can treat severe combined immunodeficiency caused by adenosine deaminase deficiency.

Original source:

Donald B.

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.

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