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immune response anytime a foreign object is

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Unformatted text preview: rs: cell and nuclear membranes membranes •safety of delivery system (are viral vectors safe?) •Immune response - Anytime a foreign object is introduced into into human tissues, the immune system is designed to attack the invader. The risk of stimulating the immune system in a way way that reduces gene therapy effectiveness is always a potential risk. •treatment usually temporary – needs to be repeated every ll few weeks/months for the whole lifetime • Many multiple gene disorders – not suited for gene multiple gene disorders not suited for gene therapy therapy (which is best for single gene disorder) 21 http://learn.genetics.utah.edu/units/genetherapy/gtenhance/ 22 GermGerm-line gene therapy 23 http://learn.genetics.utah.edu/units/genetherapy/gtenhance/ 24 What What is the Goal of Stem Cell Research? •Why don't we live forever? Because we get sick? Because we get old? we get old? Because we get hurt and can't heal? •Stem cells form the basic building building materials for the human body. This makes them good candidates for restoring tissues that have been damaged by that have been damaged by injury injury or disease. •For decades, researchers have bee study been studying the biology of stem cells to figure out how development works and to find new ways of treating health problems problems. 25 Stem cell technology Stem cell technology In a fertilized egg – the first cells are all pluripotent (i (i.e. capable to becoming any kind of cell) ki •At a later stage they differentiate to specialize (for certain certain tissues) – after which there is no turning back •Undifferentiated cells are called stem cells, and in Undifferentiated cells are called stem cells, and in those those in the embryo are called embryonic stem cells 26 So So – these pluripotent stem cells – can differentiate to become any type of cells that differentiate to become any type of cells that are are required Th th Thus they can be manipulated in vitro (e.g. genes introduced) before being replaced in to th the body – either to correct defective mutations, or to replace damaged cells. Oth Other types of stem cells also exist which have partially differentiated so are capable of becoming a number of types of cell in a 27 particular tissue • Blastocyst embryonic stem cells. These pluripotent embryonic stem cells These pluripotent cells cells are thought to be the most versatile type of stem cell, because they can become almost any type of cell in the body. • Fetal stem cells. The pluripotent stem cells found in fetal tissue (the tissue needed for the treatment came from prematurely terminated human fetuses or late-stage lateembryos)are embryos)are biologically excellent candidates for the treatment. • Umbilical cord blood stem cells. The multipotent stem cells from umbilical cord blood have the potential to turn ll bl th into many different types of cells, but their natural fate is to become blood and immune cells. • Adult stem cells. There are many different types of Ad T...
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This note was uploaded on 03/21/2013 for the course GEK 1527 taught by Professor Tan during the Winter '11 term at National University of Singapore.

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