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.
