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SUMMARY OF OVERVIEW OF PRENATAL GENE AND STEM CELL THERAPY

GENE THERAPY
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Genes
o are specific sequences of bases that encode instructions on how to make proteins
genetic disorders
o single gene disorders
sickle cell, cystic fibrosis, tay-sachs dx
o chromosome disorders
o multifactorial inheritance disorder
heart dxs, most cancers and Alzheimers dx

Gene Therapy
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defined as
o nucleic-acid based treatment, OR transfer of DNA/RNA to somatic target cells in
the intention to treat serious illness
involves correcting faulty genes via:
o insertion of a normal gene into a gene locus to replace a non-functional gene (MOST
common approach)
o swapping abnormal gene for a normal gene through homologous recombination
o Selective Reverse mutation of an abnormal gene to make it normal
o Alteration of the regulation of a particular gene
How it works
o A vector is used to insert normal gene into the gene locus e.g. viruses
o Non-viral options
Direct introduction of therapeutic DNA into target cells.
Using Liposome to carry therapeutic DNA to target cells
Problems with gene therapy
o Short-lived nature of gene therapy
o Immune response to the gene introduced
o Problems with viral vectors (toxicity, immune and inflammatory response, 0
o Multi-gene disorders e.g. heart dx, Alzheimers dx, Diabetes,
Forms
o Somatic gene therapy
done in the adult cells of people known to have the disease
here new genes are introduced to the body
o Germline gene therapy
done in egg and sperm cells i.e. human germ line is modified, conferring
heritable modification to the off sprig
However, it is not permitted in any county, on the basis that it is unethical
Types
o Postnatal gene therapy
o RNAi
Postnatal gene therapy
o Vectors are used for insertion within genome, thus imparting expression of newly
incorporated gene, and so-called gain of function
Viruses (retroviruses)
Non-viral vectors - - - have a decreased transfer (of gene) efficiency
o Types
Gene Replacement
Gene Addition

E.g. has been used to introduce the so-called suicide gene into
cancer cells

Application
Have been used in treating children with X-linked SCID
Fetal gene therapy, i.e. Prenatal or in utero gene therapy
o Benefits of prenatal gene therapy
early phenotypic correction
Demonstration of long-term postnatal therapeutic protein production
RNA interference
o Using small interfering RNA, or siRNA to destroy or down-regulate or interfere
with genes
o It principal issue is making the siRNA to deliver gene
Adverse effects of gene therapy
o Vector induced oncogenesis
o Developmental aberrations
o Germline transfer of transgenic DNA sequences
o New gene inserted into wrong location detrimental immunological effects
o Vector viruses can infect more than one type of cells
ETHICS OF GENE THERAPY
o HUGO Ethics committee statement on Gene therapy research -1999
Germ-line cell gene therapy avoided, only confronts somatic cell therapy
Application of Gene therapy
o Treatment of Type I Diabetes
o Treatment of X-linked SCID (severe combined immunodef.)
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STEM CELLS
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Definition
o Stem cells are Proliferative cells with the capability for self-maintenance
o