Fundamentals of Cellular and Molecular Biology

Application of Molecular Biology in Gene Therapy

Author(s): Maheen Shafiq *

Pp: 121-131 (11)

DOI: 10.2174/9789815238037124010012

* (Excluding Mailing and Handling)


 Gene therapy treats genetic and acquired diseases by introducing functional genes into cells to replace or correct defective genes. The field of molecular biology has played a significant role in the development of gene therapy, providing tools and techniques to manipulate and analyze genes and their expression. One of the main challenges in gene therapy is the efficient delivery of therapeutic genes to the target cells. Molecular biology has provided a range of vectors, such as viruses and plasmids, that can be used to deliver genes to cells and methods to modify these vectors to improve their efficacy and safety. Molecular biology has also contributed to the development of gene editing technologies, such as CRISPR-Cas9, which can be used to correct or modify genes at the genomic level. This approach can potentially treat genetic disorders by targeting the underlying genetic mutations. In addition, molecular biology has facilitated the development of methods to regulate gene expressions, such as gene silencing and RNA interference, which can be used to turn off genes that cause disease. Despite the progress made in gene therapy, many challenges remain to be addressed, including ensuring the safety and efficacy of gene therapy approaches. Continued advancements in molecular biology are critical for developing safe and effective gene therapies for treating genetic and acquired diseases.

Keywords: Functional genes, Genetic disorders, Gene editing, Gene expression, Defective genes, Gene silencing, Gene therapy, Molecular biology, Plasmids, Vectors, Viruses.

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