Gene and Cell Therapy

What is Gene Therapy?

• The introduction, removal or change in genetic material in
the cells of a patient to treat an inherited or developed
disease.
• Typically, genetic material, such as a working copy of a
gene, is transferred into the target cell using a vector (i.e.,
non-virulent virus)
• Once in the cell, a working copy of the gene will help make
functioning proteins despite the presence of a faulty gene. 
• Achieving the normal expression and function of proteins
makes a big impact on our overall health.
 Types of Gene Therapy
1. Gene Addition
 involves inserting a new copy of a gene into the target
cells to produce more of a protein. 
2. Gene Correction
 can be achieved by modifying part of a gene using
recently-developed gene editing technology (e.g.
CRISPR/cas9, TALEN or ZFN) to remove repeated or faulty
elements of a gene, or to replace a damaged or
dysfunctional region of DNA
 The goal of gene correction is to produce a protein that
functions in a normal manner instead of in a way that
contributes to disease.
 Types of Gene Therapy
3. Gene Silencing
 prevents the production of a specific protein by
targeting mRNA for degradation so that no protein is
produced.
4. Reprogramming
 involves adding one or more genes to cells of a specific
type to change the characteristics of those cells. 
5. Cell elimination
 used to destroy malignant (cancerous) tumor cells, but
can also be used to target overgrowth of benign (non-
cancerous) tumor cells. 
 What is Cell Therapy?
• The transfer of cells into a patient with the
goal of improving a disease
• An example of this approach is CAR-T cell
therapy. A patient’s T cells, which are a kind of
immune cell, are changed in a lab by using a
vector to add a gene that changes cells in a
way that enables them to recognize and attack
cancer cells.
https://www.asgct.org/ASGCT/media/about/
Infogrpahic-Defining_The_Approaches-1.png
 Types of Cell Therapy
1. Embryonic stem cells (ESCs)
o These are pluripotent stem cells (can give rise to all the
types of cell) derived from embryos.
2. Induced pluripotent stem cells (iPSCs)
o A differentiated adult (somatic) cell, such as a skin cell is
reprogrammed to return to a pluripotent state. 
3. Nuclear transfer embryonic stem cells (ntESCs)
o These pluripotent cells are produced by transferring the
nucleus from an adult cell obtained from the patient to
an oocyte (egg cell) obtained from a donor.  
o Dolly, the sheep, is a product of this technique
A stem cell
https://www.nationalmssociety.org/NationalMSSociety/media/
MSNational/Research/stemcell201607.jpg?ext=.jpg
Dolly, the sheep, was the poster child of modern
biotechnology. It was the first mammal to have been
successfully cloned from an adult cell.
https://upload.wikimedia.org/wikipedia/commons/thumb/6/6e/
Dolly_face_closeup.jpg/220px-Dolly_face_closeup.jpg
http://www.quotemaster.org/images/ea/eaa2132b4bfd30f6cbd9acd3e55a9b2e.png
 Types of Cell Therapy
4. Hematopoietic stem cells (HSCs)
o are multipotent blood stem cells that give rise to all types of blood
cells.
5. Immune cell therapy
o The cells can be removed from the body, isolated from a mixed cell
population, modified and then expanded before return to the body.
o CAR-T cell therapy is an example.
6. Other stem cell sources:
o Mesenchymal
o Neural
o Epithelial
 Stem Cell Therapy in the Philippines

• Administrative Order No. 2013-0012.

– prohibits and restricts the creation, importation,
promotion, marketing and use of stem cell
therapies from embryonic, aborted fetal, and
genetically altered, animal and plant stem cells.