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Gene transfer in human lymphocytes using a vector based on adeno-associated virus.
C A Muro-Cacho1, R J Samulski, D Kaplan
1Institute of Pathology, Case Western Reserve University, Cleveland, Ohio 44106.
Journal of Immunotherapy : Official Journal of the Society for Biological Therapy
|May 1, 1992
Summary
Adeno-associated virus vectors offer a safe method for gene transfer in human lymphocytes. The integrated vector remained stable, showing no adverse effects, making it a promising alternative for gene therapy.
Area of Science:
- Molecular Biology
- Virology
- Gene Therapy
Background:
- Adeno-associated virus (AAV) is a nonpathogenic parvovirus known for site-specific integration into human chromosome 19.
- Gene therapy aims to introduce genetic material into cells to treat diseases, with viral vectors being a common delivery method.
Purpose of the Study:
- To develop and assess an adeno-associated virus-based vector for gene transfer into human lymphocytes.
- To evaluate the safety and stability of the integrated vector in T-cell clones.
Main Methods:
- Utilized the inverted terminal repeats of AAV to create a gene transfer vector.
- Introduced a neomycin resistance gene into human T-cell clones.
- Assessed functional properties of infected clones and tested vector rescue/replication with adenovirus superinfection.
Main Results:
- Stable integration of the neomycin resistance gene into human T-cell clones was achieved.
- No detectable alterations in the functional properties of the infected T-cells were observed.
- The AAV vector did not rescue or replicate upon adenovirus superinfection, indicating vector stability.
Conclusions:
- Adeno-associated virus vectors can be stably integrated into human lymphocytes for gene transfer.
- The vector demonstrated a high level of safety due to its inability to replicate under superinfection conditions.
- AAV-based vectors represent a viable alternative to retroviral vectors for lymphocyte gene therapy.