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Gene Therapy

Gene therapy involves inserting a normally functioning copy of a mutated gene into affected cells to restore normal function. It has been experimentally successful in treating some diseases like melanoma, immunodeficiencies, and sickle cell anemia. Viruses are commonly used as vectors to deliver the healthy gene into cells. Different viral vectors have advantages and disadvantages related to their ability to integrate the gene into the host genome safely and effectively deliver the gene to the desired cells. Researchers are working to develop improved viral vectors to address past safety issues.

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1K views11 pages

Gene Therapy

Gene therapy involves inserting a normally functioning copy of a mutated gene into affected cells to restore normal function. It has been experimentally successful in treating some diseases like melanoma, immunodeficiencies, and sickle cell anemia. Viruses are commonly used as vectors to deliver the healthy gene into cells. Different viral vectors have advantages and disadvantages related to their ability to integrate the gene into the host genome safely and effectively deliver the gene to the desired cells. Researchers are working to develop improved viral vectors to address past safety issues.

Uploaded by

patricia anne
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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Download as PPT, PDF, TXT or read online on Scribd
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Gene Therapy

What is Gene Therapy?


• Gene therapy is a
treatment or cure for
disorders caused by
mutated genes.
• It involves adding a
normally functioning
copy of the gene(s) to
affected cells to restore
normal function.
What is Gene Therapy
• Germline gene therapy would be the
permanent transfer of a gene into
sperm or egg cells.
– Future generations would be “cured”.
• Somatic cell (body cell) gene therapy
is ideally only the transfer of genes
to the affected cells.
Gene Therapy Successes
• Although no gene therapies have
been approved by the FDA for sale,
some diseases have been
experimentally successful:
– Melanoma (skin cancer)
– Severe Combined Immunodeficiencies
– Hereditary Blindness
– Sickle Cell Anemia
How is it done?
Viral Vector Carrying Healthy Gene

Cell with mutated Vector inserts New gene in the


gene(s) healthy gene into cell along with
cell original genes
Functional proteins are created from the therapeutic gene
causing the cell to return to a state.
Gene Therapy
To design and carry out a gene therapy
treatment, a researcher must:
1. Identify the gene(s) responsible for the
disorder.
2. Make copies of the normal gene.
3. Insert the copies into vectors.
4. “Infect” the affected cells with the vectors.
5. Activate the gene so that transcription and
translation take place.
Viruses as Vectors
• Replicate by inserting their DNA into
a host cell
• Gene therapy can use this to insert
genes that encode for a desired
protein to create the desired trait
• Four different types
– Adenovirus
– Adeno-Associated Virus (AAV)
– Retrovirus
– Herpes Simplex Virus (HSV)
Vector Advantages and Disadvantages
• Adenovirus
+ Infects many cell types
− Does not integrate into host genome and can be lost.
• Retrovirus
+ Integrates into host genome and cannot be lost
− Integrates into host genome and can cause cancer
• Adeno-Associated Virus (AAV)
+ Integrates into host genome and cannot be lost
− Difficult to work with.
• Herpes Simplex Virus (HSV)
+ DNA stays in nucleus without integrating into host
genome.
− Only infects cells of the nervous system.
Gene Therapy
Disappointments
• In 1999 a boy died due to an
immune response to an adenovirus
gene therapy vector.
• Four children have developed
cancer due to a retrovirus gene
therapy vector
Research: Building a better Vector

Adenovirus
HSV-like virus
shell delivers
protein tethers DNA
genes
to chromosome to
keep genes in cell
without integration

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