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Gene therapy: basic concepts and recent advances
1Department of Pathology, University of Pennsylvania School of Medicine, Philadelphia 19104, USA.
Abstract:
After years of preliminary in vitro and in vivo modeling, the first human gene therapy clinical trial was initiated approximately 5 years ago. The experience gained from this trial, and the others that have come after it, has shown that effective and efficient human gene therapy remains a tantalizing but elusive goal. Nevertheless, interest in this area of therapeutics continues to grow and over 100 gene therapy protocols have now been submitted to the Recombinant DNA Advisory Committee ("RAC") of the National Institutes of Health for approval. The goal of these protocols vary, but include correction of inherited gene defects, engineering new traits into cells such as diminished chemosensitivity in the case of bone marrow cells, or, in the case of tumor cells, expression of new immunophenotypes. Marking cells with a gene whose function can easily be detected, thereby rendering the cell traceable in vitro and in vivo is also the goal of several clinical trials. At the same time, different means of gene transfer are been investigated for the efficiency with which they deliver "therapeutic nucleotides" into their cellular targets. In this review, we recount some of the major developments that have occurred in these various areas and discuss their impact on progress in the field.
Insights
Human gene therapy trials show promise but face challenges in effectiveness and efficiency. Ongoing research focuses on correcting genetic defects, enhancing cell traits, and improving gene transfer methods for therapeutic applications.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- Human gene therapy clinical trials have been ongoing for approximately five years.
- Despite preliminary modeling, achieving effective and efficient human gene therapy remains a significant challenge.
- Over 100 gene therapy protocols have been submitted for approval, indicating growing interest in the field.
Purpose of the Study:
- To review major developments in human gene therapy.
- To discuss the impact of these developments on the progress of the field.
- To highlight diverse goals of gene therapy protocols, including disease correction and cell engineering.
Main Methods:
- Review of in vitro and in vivo modeling data.
- Analysis of clinical trial outcomes and submitted protocols.
- Investigation of various gene transfer methods for therapeutic nucleotide delivery.
Main Results:
- Gene therapy aims to correct inherited defects, engineer new cellular traits (e.g., diminished chemosensitivity), and modify tumor cell immunophenotypes.
- Cellular tracking in vitro and in vivo is a key goal, achieved by marking cells with detectable genes.
- Research is actively exploring efficient gene transfer techniques to deliver therapeutic nucleotides.
Conclusions:
- Human gene therapy has progressed significantly, with numerous protocols submitted and diverse applications explored.
- While challenges in efficacy and efficiency persist, ongoing research in gene transfer and therapeutic applications continues to advance the field.
- The field shows a strong trajectory with continued interest and development in gene therapy strategies.