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Current developments in the design of onco-retrovirus and lentivirus vector systems for hematopoietic cell gene

Sebastian Brenner1, Harry L Malech

  • 1National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA. sbrenner@nih.gov

Biochimica Et Biophysica Acta
|April 5, 2003
PubMed

Insights

Gene therapy using oncoretrovirus vectors has shown success in treating severe combined immunodeficiency (SCID). However, challenges remain, including insertional mutagenesis risks in gene transfer therapies.

Area of Science:

  • * Molecular Biology
  • * Immunology
  • * Oncology

Background:

  • * Oncoretrovirus vectors have been widely used in gene therapy for genetic diseases and cancer.
  • * Early retroviral vectors faced limitations in transduction and transgene expression efficiency.
  • * Cellular biology and immunology posed significant challenges to effective gene therapy.

Purpose of the Study:

  • * To review the progress and challenges in oncoretrovirus-mediated gene therapy.
  • * To highlight advancements in overcoming vector limitations.
  • * To discuss the implications of recent clinical successes and safety concerns.

Main Methods:

  • * Review of clinical gene therapy trials utilizing oncoretrovirus vectors.
  • * Analysis of data on transduction and transgene expression rates.
  • * Examination of case studies for X-linked severe combined immunodeficiency (X-SCID) and adenosine deaminase (ADA)-deficient SCID.

Main Results:

  • * Successful functional correction of immune deficiencies in X-SCID and ADA-SCID patients through ex vivo gene therapy.
  • * Demonstrated prolonged clinical benefits in treated patients.
  • * Identified insertional mutagenesis as a significant adverse event in two X-SCID patients.

Conclusions:

  • * Oncoretrovirus vectors have shown promise in treating genetic immune disorders.
  • * Ongoing development of novel retroviral vectors aims to improve efficacy and safety.
  • * Insertional mutagenesis remains a critical safety concern requiring further investigation and mitigation strategies.

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