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Genetically engineered intracellular single-chain antibodies in gene therapy

Guadalupe Bilbao1, Juan Luis Contreras, David T Curiel

  • 1University of Alabama at Birmingham, Department of Medicine, Pathology and Surgery, Gene Therapy Center, 35294, USA.

Molecular Biotechnology
|October 31, 2002
PubMed

Insights

Gene therapy offers targeted cancer treatment by addressing molecular and genetic causes. This article details methods for developing single-chain intracellular antibodies (scFv) for mutation compensation strategies.

Area of Science:

  • Oncology
  • Molecular Biology
  • Genetics

Background:

  • Cancer's molecular basis is increasingly understood, enabling targeted therapies.
  • Genetic lesions driving cancer progression are being identified.
  • Aberrant gene expression patterns are characteristic of malignant disorders.

Purpose of the Study:

  • To review gene therapy as a rational approach for inherited and acquired diseases.
  • To detail strategies for mutation compensation, including targeting oncogenes and tumor suppressor genes.
  • To provide practical procedures for generating single-chain intracellular antibodies (scFv).

Main Methods:

  • Review of mutation compensation strategies: DNA (triplex), mRNA (antisense, ribozyme), and protein (intracellular single-chain antibodies).
  • Focus on augmenting tumor suppressor gene expression.
  • Detailed protocol for generating scFvs against various target proteins.

Main Results:

  • Elucidation of the genetic basis of diseases supports gene therapy.
  • Development of practical protocols for scFv generation.
  • Demonstration of scFv utility in targeting specific proteins.

Conclusions:

  • Gene therapy is a promising approach for treating genetic and acquired diseases, including cancer.
  • Single-chain intracellular antibodies (scFv) are a key tool in mutation compensation strategies.
  • The described protocols facilitate the development of targeted molecular interventions for cancer therapy.

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