Wh •at is gene therapy?
Gences (carried on our chromosomes) are specific sequences of bases that contain instructions on how to
makoe proteins.Although genes get a lot of attention, it is the proteins which the genes produce that
permorm most life functions and make up majority of cell structures. When genes are altered, they cannot
pro/duce proteins that carry out their normal functions, resulting in genetic disorders.
Gente therapy involves the insertion, alteration, or removal of genes within cells and biological tissues to
treet any genetic disorders that have been caused by abnormalities in chromosomes. It is a technique for
cormecting defective genes that are responsible for disease development.
Ways available to researchers for correcting faulty genes:
l• The first form of gene therapy involves the insertion of a functional gene.Anormal gene may be
l inserted into a nonspecific location within the genome to replace a nonfunctional gene. This
- approach is most common amongst researchers.
• The second form of gene therapy involves switching an abnormal gene with a normal one.An
o abnormal gene can be swapped for a normal gene through homologous recombination.
-• The third approach involves directly correcting the mutation. The abnormal gene is repaired to
a return to its normal function. This is done through selective reverse mutation.Areverse mutation
d causes a gene to overt back to its original function.
What is stem-cell research?
Stem cells are biological cells found in all multicellular organisms. They can divide and differentiate into
diverse specialized cell types and self-renew to produce more stem cells. In humans the two types of stem
cells include embryonic cells, which are isolated from the inside of blastocysts, and adult stem cells
whi•h are found in various tissues of the body. Embryonic stem cells can differentiate into all types of
spetialized cells such as skin cells, nerve cells, blood cells, etc.Adult stem cells act as a repair system for
the body, replenishing adult tissues.
Stem cells are important to biomedical researchers because they can be used to generate any type of
specialized cell in the human body. Embryonic stem cells and adult stem cells are extracted so that
resxarchers can study their development to potentially allow for them to treat diseases. Stem cells are
grozn in laboratories where tests are carried out to investigate fundamental properties of the cells.
Embryonic stem cells are extracted from the egg at the blastocyst stage of development. They can also be
generated through “therapeutic cloning”, which is cloning of the embryo. The extraction process destroys
the embryo, which raises ethical concern. It has been proposed that surplus embryos created for in vitro
fertilization could be donated with consent and used for the research.
ThrYe main ways in which adult stem cells can be extracted.
1) pone marrow -- requires extraction by harvesting (drilling into bone)
2)Adipose tissue (lipid cells) -- requires extraction by liposuction
3) Blood, -- requires extraction through pheresis. Blood is drawn from the donor and passed through a
machine that extracts the stem cells and returns other portions of the blood back to the donor.
G What are the possible social benefits of applications of stem-cell research?
There are many health benefits of stem-cell research. Researchers believe that stem cells can one day be
used as replacement cells to treat a variety of diseases.Anumber of adult stem cell therapies already
exist, including bone marrow transplants which are used to treat leukemia. In the future, technologies
derived from stem cell research will allow to treat a wider range of diseases including cancer, Parkinson's
disease, spinal cord injuries, Amyotrophic lateral sclerosis, multiple sclerosis, muscle damage, spinal
cord damage, and many other conditions illustrated in the image.
The social benefits of stem cell research will be countless.As the science develops, society will discover
many improvements in human life.
•Quality of life: the quality of life will be improved because many diseases could be treated, resulting in a
longer life expectancy and better overall health.
•Healthier work force: Due to cures being ava