University of Science andTechnology 0f Southern Philippines

College of Science and Technology Education

First Semester SY 2018-2019

Out Activity :

What do you learn in today's lesson? Why are Stem Cells are important?

Stem cells are important for living organisms for many reasons. In the 3- to 5-day-old embryo,

called a blastocyst, the inner cells give rise to the entire body of the organism, including all of the

many specialized cell types and organs such as the heart, lung, skin, sperm, eggs and other

tissues. In some adult tissues, such as bone marrow, muscle, and brain, discrete populations of

adult stem cells generate replacements for cells that are lost through normal wear and tear,

injury, or disease. Given their unique regenerative abilities, stem cells offer new potentials for

treating diseases such as diabetes, and heart disease.

 University of Science andTechnology 0f Southern Philippines

College of Science and Technology Education

First Semester SY 2018-2019

Cornell Notes on Gene Therapy and Stem Cell Therapy

It seeks to alter genes to correct genetic defects

What is Gene Therapy? in order to prevent or cure genetic diseases.

1. Somatic gene therapy

 insertion of therapeutic DNA
into body cells
 effects of the therapy are
confined to the individual being
What are the two basic types of Gene Therapy? treated.
2. Germline gene therapy
 modification of the genes inside
germ cells (sperm or ova)
 alters the genome of future
generations to come.

1. Ex vivo
 transfer of genes in cultured
What are the two approaches of Gene Therapy? cells and will be reinjected to the
body of the patient.
2. In vivo
 introduction of therapeutic gene
into the vector injected directly
to the body.
 1. Treatment begins with cells being
removed from an infected person. These
cells are then grown and multiplies in a
laboratory.

2. Therapeutic gene will be introduced into

the cultured cells using a virus called
How does ex-vivo gene therapy? Vector. The virus’s DNA is taken out
and replaced with the one that treats the
patient.

3. The genetically transformed cells are

selected and reinjected into the patient’s
body.

1. Involves direct delivery of the

therapeutic gene into the target cells of a
How does in-vivo gene therapy? particular tissue of a patient
2. Can be carried out using viral or non-
viral vectors
3. Success depends on:
a. efficiency of the uptake of the
therapeutic gene by the target
cells
b. intracellular degradation of the
gene and its uptake by nucleus
c. the expression capability of the
gene
 It refers to carrier particles or molecules used to
deliver genes. This includes :
What is vector ? 1. Viral vectors
2. Non-Viral vectors

 Replicate by inserting their DNA into a

What is viruses ?
What are the four different types of viruses ?
host cell.
 Gene therapy can use this to insert
genes that encode for a desired protein
to create the desired trait.
1. Retroviruses
 Created double stranded DNA
copies from RNA genome
2. Adenoviruses
 Double stranded DNA genome
that causes respiratory,intestinal
and eye infections in humans
3. Adeno-associate virus
 Small, single stranded DNA that
insert genetic material at a
specific point on chromosome
19
4. Herpes Simplex Viruses
 Double stranded DNA viruses
that infect neurons
  Short Lived
 Immune Response
 Viral Vectors
What are the problems of gene therapy ?  Multigene Disorders
 May induce a tumor if integrated in a
tumor suppressor gene because
insertional mutagenesis

A cell that has the ability to continuously

What is Stem Cell? divide and differentiate (develop) into various
other kind(s) of cells/tissues

 Blank cells’ (unspecialized)

 Capable of dividing and renewing
themselves for long periods of time
What are the Stem Cell Characteristics? (proliferation and renewal)
 Have the potential to give rise to specialized
cell types (differentiation)

1. Tipotent
 Each cell can develop into new
individual
2. Pluriotent
What are the types of Stem Cells?  Cells can form any (over 200)
cell types
3. Multipotent
 Cells differentiated, but can
form a number of other tissues

What are Two main sources of stem cells?
What is the Stem Cell Applications?
Why is stem cell research so important to ll of
us?
1. Embryonic Stem cells
 derived from a four- or five-day-
old human embryo that is in the
blastocyst phase of development
2. Adult Stem Cells
 found in tissues such as the
brain, bone marrow, blood,
blood vessels, skeletal muscles,
skin, liver and umbilical cord.
• Tissue repair
• Cancers
• Autoimmune diseases
 Stem cells can replace diseased or
damaged cells
 Stem cells allow us to study
development and genetics
 Stem cells can be used to test different
substances (drugs and chemicals)
SUMMARY :

 Gene Therapy seeks to alter genes to correct genetic defects in order to prevent or cure

genetic diseases.

 The two (2) types of gene therapy are Somatic gene therapy and Germline gene therapy.

 The two approaches of gene therapy are Ex-vivo and In-vivo.

 Viruses are replicate by inserting their DNA into a host cell.

 The four different types of virus are Retroviruses, Adenoviruses, Adeno-associated

viruses and Herpes Simples Viruses.

 The problems with gene therapy are Short Lived, Immune Response, Viral Vectors,

Multigene Disorders and may induce a tumor if integrated in a tumor suppressor gene

because insertional mutagenesis.

 Stem Cells are cell that has the ability to continuously divide and differentiate (develop)

into various other kind(s) of cells/tissues.

 There are three (3) typess of stem cell which are Tipotent, Pluripotent and Multipotent.

 The two main sources of stem cells are Embryonic stem cells and Adult stem cells.