Evolution of Gene Therapy, Historical Perspective
Harry L Malech1, Elizabeth K Garabedian2, Matthew M Hsieh3
1Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, 10 Center Drive, MSC1456, Bldg 10, Rm 5-3750, Bethesda, MD 20892-1456, USA.
Hematology/Oncology Clinics of North America
|June 30, 2022
Summary
Gene therapy harnesses DNA to modify cells, using engineered viruses to deliver genetic material into human hematopoietic stem cells (HSC). Advances in HSC culture and clinical trials have improved gene therapy safety and efficacy.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- The concept of gene therapy originated from understanding DNA's role in heredity and bacterial transformation.
- Early research on oncogenic viruses suggested their potential for genetic material delivery into mammalian cells, including human hematopoietic stem cells (HSC).
Purpose of the Study:
- To provide a historical overview of the key discoveries and technological advancements that have shaped current gene therapy targeting HSC.
Main Methods:
- Tracing the conceptual evolution from DNA's discovery to viral vector engineering.
- Highlighting the development of essential technologies for HSC procurement, purification, and culture.
- Reviewing the role of clinical trials in assessing efficacy and safety.
Main Results:
- Successful gene therapy for HSC requires engineered vectors for safe transduction.
- Development of efficient methods for HSC manipulation and patient conditioning is crucial.
- Clinical trials have been pivotal in advancing gene therapy efficiency and safety.
Conclusions:
- Gene therapy for HSC is a convergence of multiple scientific and technological breakthroughs.
- Continued research and clinical evaluation are essential for further progress in the field.
Keywords:
ApheresisCD34+ HSCGamma retrovirus vectorGene editingHematopoietic stem cellsInsertional mutagenesisLentiviral vectorTransductionMore Related Videos
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