Welcome to Gene Therapy
At the forefront of medicine, Gene Therapy brings you the latest
research into genetic and cell-based technologies to treat disease.
It also publishes Progress & Prospects reviews and News and
Commentary articles, which highlight the cutting edge of the field.
Genes, which are carried on chromosomes, are the basic physical
and functional units of heredity. Genes are specific sequences
of bases that encode instructions on how to make proteins. Although
genes get a lot of attention, it’s the proteins that perform
most life functions and even make up the majority of cellular
structures. When genes are altered so that the encoded proteins
are unable to carry out their normal functions, genetic disorders
can result.
Gene therapy is a technique for correcting
defective genes responsible for disease development. Researchers
may use one of several approaches for correcting faulty genes:
A normal gene may be inserted into
a nonspecific location within the genome to replace a nonfunctional
gene. This approach is most common. An abnormal gene could be swapped
for a normal gene through homologous recombination.
The abnormal gene could be repaired
through selective reverse mutation, which returns the gene to its
normal function.
The regulation (the degree to which
a gene is turned on or off) of a particular gene could be altered.
Gene therapy is the insertion of genes into an individual's cells
and tissues to treat a disease, and hereditary diseases in which
a defective mutant allele is replaced with a functional one. Although
the technology is still in its infancy, it has been used with some
success. Antisense therapy is not strictly a form of gene therapy,
but is a genetically-mediated therapy and is often considered together
with other methods. |
