Activity Gene Therapy

FAMILY NAME: SANDALO GIVEN NAME: MA. ANDREA M.
I B
READING EXERCISE:
1. Describe gene therapy and its two types:

a. Somatic Gene Therapy
b. Germline Gene Therapy
2. What health condition do you think urgently needs to be treated by gene therapy? Justify your answer.
ANSWER HERE:
1.
 Gene therapy is when DNA is introduced into a patient to treat a genetic disease. The new DNA usually contains a functioning gene to
correct the effects of a disease-causing mutation.
o Gene therapy uses sections of DNA? (usually genes?) to treat or prevent disease.
o The DNA is carefully selected to correct the effect of a mutated gene that is causing disease.
o The technique was first developed in 1972 but has, so far, had limited success in treating human diseases.
o Gene therapy may be a promising treatment option for some genetic diseases?, including muscular dystrophy? and cystic
fibrosis?.
There are two different types of gene therapy depending on which types of cells are treated:
 Somatic gene therapy: transfer of a section of DNA to any cell of the body that doesn’t produce sperm or eggs. Effects of gene therapy
will not be passed onto the patient’s children.
 Unlike germline therapy, somatic gene therapy involves the insertion of therapeutic DNA into body cells, rather than germ cells or
gametes. This means that any effects of the therapy are confined to the individual being treated and are not inherited by future
offspring.
 The field of somatic gene therapy is surrounded by fewer ethical issues as compared to germline gene therapy. While this may be true,
this therapeutic approach remains in the early stages of development.
 The first hurdle in somatic gene therapy is the successful incorporation of the new gene into the genome. In fact, integrating the
modified gene into the wrong part of the DNA could induce rather than prevent disease. In addition to requiring the desired gene
needs to be expressed, the gene expression of the new gene needs to be regulated in order to prevent over-expression that could also
trigger disease.
 Germline gene therapy: transfer of a section of DNA to cells that produce eggs or sperm. Effects of gene therapy will be passed onto the
patient’s children and subsequent generations.
 Germline therapy involves the modification of the genes inside germ or gamete cells, which include sperm or ova. Germline therapy
would therefore be administered during reproduction, where the modified gamete cells fuse to form a zygote. Once fused together,
the zygote divides and passes on the modified gene to all other cells of the body during the development of offspring. In this way,
germline therapy alters the genome of future generations to come.
 Although theoretically, germline therapy could counteract hereditary diseases, jurisdictions in various countries such as Switzerland,
Australia, and Germany prohibit the use of germline therapy due to fears on the unknown risks of this therapy and whether it causes
any long-term effects in future generations. Germline therapy is also extremely expensive, which further limits its practical use.
2
 Gene therapy attempts to treat cancer or boost the body's ability to combat disease by replacing a defective gene by adding a new
gene. Gene therapy carries promise in the treatment of cancer, cystic fibrosis, heart disease, diabetes, hemophilia, and AIDS, among
other illnesses.
 Gene Therapy was initially meant to introduce genes straight into human cells, focusing on diseases caused by single-gene defects, such
as cystic fibrosis, hemophilia, muscular dystrophy (see video 2) and sickle cell anemia (see also Wiley database on indications addressed
by gene therapy clinical trials). Three types of diseases for gene therapy can be distinguished:
o Monogenic disorders, single locus (gene) is defective and responsible for the disease, 100% heritable. Examples: Sickle cell
anemia, Severe Combined Immunodeficiency (ADA-SCID / X-SCID), Cystic fibrosis, Hemophilia, Duchenne muscular dystrophy,
Huntington’s disease, Parkinson’s, Hypercholesterolemia, Alpha-1 antitrypsin, Chronic granulomatous disease, Fanconi Anemia
and Gaucher Disease.
o Polygenic disorders, multiple genes involved, disease may be dependent on environmental factors and lifestyle. Examples: Heart
disease, Cancer, Diabetes, Schizophrenia and Alzheimer’s disease.
o Infectious diseases, such as HIV.

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FAMILY NAME: SANDALO GIVEN NAME: MA. ANDREA M.

1. Describe gene therapy and its two types:

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