Gene therapy for adenosine deaminase deficiency

Robertson Parkman, Kenneth Weinberg, Gay Crooks, Jan Nolta, Neena Kapoor, Donald Kohn

Research output: Contribution to journalArticlepeer-review

27 Scopus citations


The clinical gene therapy trials for adenosine deaminase (ADA) deficiency have defined both the potential benefits and the present limitations of gene therapy with hematopoietic stem cells (HSC). Current clinical results indicate that (a) both umbilical cord blood and neonatal bone marrow HSC can be transduced with murine retroviral-based vectors, (b) the transduced HSC can engraft in nonmyeloablated patients, (c) the frequency of HSC transduction/engraftment is low (1/10,000), (6) an in vivo selective advantage can exist for transduced T lymphoid progeny, and (e) the transduced ADA gene is not expressed in nondividing T lymphocytes. Improving the clinical results of gene therapy for ADA deficiency and other genetic diseases involving HSC will require (a) developing new vectors that express the transduced gene in nondividing cells and (b) increasing the frequency of stable HSC transduction.

Original languageEnglish (US)
Pages (from-to)33-47
Number of pages15
JournalAnnual Review of Medicine
StatePublished - 2000
Externally publishedYes


  • Gene expression
  • Gene therapy
  • Hematopoietic stem cell
  • Primary immunodeficiency
  • Retroviral vector

ASJC Scopus subject areas

  • Cell Biology
  • Medicine(all)


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