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Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
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Gene therapy for inherited metabolic diseases
Berna Seker Yilmaz1,2, Sonam Gurung1, Dany Perocheau1
1Genetics and Genomic Medicine, Great Ormond Street Institute of Child Health, University College London, London, UK.
Journal of Mother and Child
|February 8, 2021
Summary
Gene therapy is revolutionizing rare disease treatment, offering new hope for inherited metabolic diseases (IMD). This review explores gene therapy strategies, vectors, and clinical applications for IMD patients.
Area of Science:
- Biotechnology and Genetic Medicine
- Rare Disease Therapeutics
Background:
- Gene therapy has advanced significantly over the past 20 years, with increasing clinical trials and market-approved products.
- Inherited metabolic diseases (IMD) are rare, often severe conditions with limited treatment options.
- Gene therapy is emerging as a transformative treatment for IMD.
Purpose of the Study:
- To review the development of gene therapy for inherited metabolic diseases (IMD).
- To detail gene therapy strategies, administration routes, vectors (viral and non-viral), and gene editing tools.
- To discuss the advantages and disadvantages of various gene therapy approaches in IMD.
Main Methods:
- Comprehensive review of gene therapy strategies and their application in IMD.
- Analysis of clinical trials utilizing lentiviral and adeno-associated viral vectors for rare diseases.
- Discussion of gene editing technologies relevant to IMD treatment.
Main Results:
- Gene therapy is a rapidly developing field with demonstrated success in rare diseases.
- Lentiviral and adeno-associated viral vectors are key tools in current gene therapy trials for IMD.
- Gene therapy offers a realistic therapeutic option for various IMD.
Conclusions:
- Gene therapy represents a significant advancement in treating rare inherited metabolic diseases.
- Understanding gene therapy technologies is crucial for physicians to effectively counsel patients.
- The field's rapid progress necessitates continuous education for healthcare providers.
Keywords:
adeno-associated virusgene therapyinherited metabolic diseaselentivirusmessenger RNAzinc finger nucleaseMore Related Videos
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