All about Gene therapy

3. James Watson in the year 1991, states that many people are
worried about the genetic changes instructions. But these genetic
instructions are product of evolution so we can adapt to certain
conditions which might no longer exists.
Since the beginning, humans understand that the peculiar
characteristics of the parents can be transmitted to their
descendents.
Genetic-scientific studies initiated in the early 1850s, when the
Austrian monk, Gregor Mendel, in a series of experiments with green
peas, described the inheritance pattern by observing the traces that
were inherited as separate units, which we know today as genes
References: Einstein (Sao Paulo). 2017 Jul-Sep; 15(3): 369–375.doi: 10.1590/S1679-45082017RB4024; PMCID: PMC5823056; PMID: 2909116

4.  Up until 1950, little was known as to the physical nature of genes, which
was when the American biochemist, James Watson, and the British
biophysicist, Francis Crick, developed the revolutionary model of the
double strand DNA.
In 1970, researchers discovered a series of enzymes that enabled the
separation of the genes in predetermined sites along the DNA molecule
and their reinsertion in a reproducible manner. These genetic advances
prepared the scenario for the emergence of genetic engineering with the
production of new drugs and antibodies
References: Einstein (Sao Paulo). 2017 Jul-Sep; 15(3): 369–375.doi: 10.1590/S1679-45082017RB4024; PMCID: PMC5823056; PMID: 2909116

5. Gene therapy is understood as the capacity for gene improvement by
means of the correction of altered (mutated) genes or site-specific
modifications that have therapeutic treatment as target.
Gene therapy seeks to treat a disease by transferring one or more
therapeutic nucleic acids to a patient’s cells or by correcting a defective
gene, for example by gene editing
References: Mol Ther Methods Clin Dev. 2016; 3: 16034; Published online 2016 May 25. doi: 10.1038/mtm.2016.34; PMCID:
PMC4879992; PMID: 27257611

6. When genes are altered so
that the encoded proteins
are unable to carry out
their normal functions,
genetic disorders can
result.

7. Genes that are flawed and do not work
properly can cause disease. Gene therapy is a
technique for correcting defective genes
responsible for disease development.
Researchers may use one of several
approaches for correcting faulty genes:
Reference: http://cisncancer.org/research/new_treatments/gene_therapy/importance.html

8. 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.
Reference:
http://cisncancer.org/research/new_treatments/gene_therapy/importance.html

10. • On September 14, 1990 at the U.S. National Institutes of Health, W.
French Anderson M.D. and his colleagues R. Michael Blaese, M.D., C.
Bouzaid, M.D., and Kenneth Culver, M.D., performed the first
approved gene therapy procedure on four-year old Ashanthi DeSilva.
Born with a rare genetic disease called severe combined
immunodeficiency (SCID)
• As of early 2007, she was still in good health, and she was attending
college. Some would state that the study is of great importance
despite its indefinite results, if only because it demonstrated that gene
therapy could be practically attempted without adverse consequences.
Reference: Foldesi B (2018), The First Human Genet Therapy; https://www.biomol.com

15. Gene Therapy has a potential risk since a gene needs a vectors to be
inserted back to the cell. The most common gene therapy vectors are
viruses because they can recognize certain cells and carry genetic
material into the cells. This technique presents the following risks:
Unwanted system reaction
Targetting the wrong cells
Infection caused by the virus
Possibility of causing a tumor
Reference: https://www.mayoclinic.org/test-genetherapy

16. 1. How can “good” and “bad” uses of gene therapy be distinguished?
2. Who decides which traits are normal and which constitute a disability or disorder?
3. Will the high costs of gene therapy make it available only to the wealthy?
4. Could the widespread use of gene therapy make society less accepting of people who
are different?
5. Should people be allowed to use gene therapy to enhance basic human traits such as
height, intelligence, or athletic ability?