Types of Disorder gene therapy can and cannot treat
can: Hemophilia,Cancer, Parkinson's disease (*), Huntington's disease*, Sickle cell anemia*
cannot: Multifactorial genetic disorders(**)
(*)Gene therapy has also been studied as a possible treatment for Parkinson's disease, a degenerative nervous system disease. Researchers have recently figured out a way to transfer genes into the brain. Viruses cannot be used as vectors because they are too big to make it across the protective membrane that separates circulating blood from brain cells (called the blood-brain barrier). So, instead of using viral vectors, researchers have used fat molecules (called liposomes) that are coated in a polymer, called polyethylene glycol (PEG), to deliver the new genes. This allows billions of copies of a gene to be inserted into the brain to calm overactive connections in the brain. Researchers are hopeful that this method may be an effective treatment for Parkinson's disease in the future
(**) Most disorders, including Alzheimer's disease and arthritis, occur when there are mutations or problems with multiple genes. These types of disorders are especially difficult to treat with gene therapy because doctors would have to correct all of the mutated genes.
http://www.naturalstandard.com/demo/demo-gp-genetherapy.asp
Explanation on what ADA-deficient SCID is
Refer to previous blog post
How gene therapy can be used to treat ADA-deficient SCID
Gene therapy for ADA-deficient SCID could be performed by introducing a normal copy of the human ADA gene into the blood forming stem cells of the patient's bone marrow by using a new type of gene delivery system (in this trial called a lentiviral vector). The gene corrected cells are then transplanted back into the patient after small dose of chemotherapy. These gene corrected stem cells can survive into the body and make lymphocytes.
Tuesday, January 1, 2013
Wednesday, December 26, 2012
I CAN't FIND THE PAPER.
Sup everyone!
How's your holidays? I, as usual, have not spent it productively for work. Things never change. Anyway, thanks wanqi for creating the blog and welcome home haha
Sadly, I can't find the paper with the instructions for the project. I'm so sorry cause now, I don't know what exactly I'm supposed to research on besides the general topic; Gene therapy. So can someone show me the light? Thanks :)
Oh, Shafiqa's in our group so Wanqi can you add her as author also? No idea how to do that cause I've never used blogger and I'm such a noob at it.
When shall we meet to complete this project? Next week?
Hope to see you all soon and enjoy the rest of the holidays :)
Aisyah
How's your holidays? I, as usual, have not spent it productively for work. Things never change. Anyway, thanks wanqi for creating the blog and welcome home haha
Sadly, I can't find the paper with the instructions for the project. I'm so sorry cause now, I don't know what exactly I'm supposed to research on besides the general topic; Gene therapy. So can someone show me the light? Thanks :)
Oh, Shafiqa's in our group so Wanqi can you add her as author also? No idea how to do that cause I've never used blogger and I'm such a noob at it.
When shall we meet to complete this project? Next week?
Hope to see you all soon and enjoy the rest of the holidays :)
Aisyah
Thursday, December 13, 2012
What is ADA?
- ADA deficiency is one form of SCID (severe combined immunodeficiency), a type of disorder that affects the immune system.
- a malfunctioning immune system leaves the body open to infection from bacteria and viruses
- The disease is caused by a mutation in a gene on chromosome 20.
- The gene codes for the enzyme adenosine deaminase (ADA). Without this enzyme, the body is unable to break down a toxic substance called deoxyadenosine.
-The toxin builds up and destroys infection-fighting immune cells called T and B lymphocytes.
READ: http://www.depressionatoz.com/related-disorders/adenosine-deaminase-deficiency.html
- ADA deficiency is an autosomal recessive disorder. Both parents need to pass the defective gene to their child in order for that child to inherit the deficiency.
- Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births
- Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare
- People afflicted with this disease often have to live in a sheltered or enclosed environment, so that they're not exposed to infectious agents.
What is gene therapy?
- Gene therapy is the use of DNA as a pharmaceutical agent to treat disease.
- DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease.
- The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene in order to replace a mutated gene.
How does gene therapy work? ** (types of vectors and delivery methods)
- do they only want for ADA? or generally all the vectors and delivery methods used?
Challenges or risks faced in the implementation of gene therapy to treat disorder
-The body’s immune system naturally attacks anything that invades it, so the body may attack the genes being introduced to it by gene therapy or may attack the carrier (vector) if it is a virus.
- viruses used to deliver genes might recover their ability to cause disease in the body.
- impossible to control where the introduced gene ends up, there is a certain risk of it ending up in the wrong place and giving rise to leukemia. This is why gene therapy is only used for serious diseases for which there is no good treatment,
- ethic problems
- Gene therapy is short-lived
In order to achieve long-term benefits of gene therapy, the genes that are inserted into the patient must remain functional after treatment. However, most types of cells that receive new genes during gene therapy eventually die and are replaced by new ones. Currently, researchers have not discovered an effective way to integrate therapeutic genes into a patient's genome. As a result, patients need to undergo multiple treatments of gene therapy in order for it to be effective.
- ADA deficiency is one form of SCID (severe combined immunodeficiency), a type of disorder that affects the immune system.
- a malfunctioning immune system leaves the body open to infection from bacteria and viruses
- The disease is caused by a mutation in a gene on chromosome 20.
- The gene codes for the enzyme adenosine deaminase (ADA). Without this enzyme, the body is unable to break down a toxic substance called deoxyadenosine.
-The toxin builds up and destroys infection-fighting immune cells called T and B lymphocytes.
READ: http://www.depressionatoz.com/related-disorders/adenosine-deaminase-deficiency.html
- ADA deficiency is an autosomal recessive disorder. Both parents need to pass the defective gene to their child in order for that child to inherit the deficiency.
- Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births
- Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare
- People afflicted with this disease often have to live in a sheltered or enclosed environment, so that they're not exposed to infectious agents.
What is gene therapy?
- Gene therapy is the use of DNA as a pharmaceutical agent to treat disease.
- DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease.
- The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene in order to replace a mutated gene.
How does gene therapy work? ** (types of vectors and delivery methods)
- do they only want for ADA? or generally all the vectors and delivery methods used?
Challenges or risks faced in the implementation of gene therapy to treat disorder
-The body’s immune system naturally attacks anything that invades it, so the body may attack the genes being introduced to it by gene therapy or may attack the carrier (vector) if it is a virus.
- viruses used to deliver genes might recover their ability to cause disease in the body.
- impossible to control where the introduced gene ends up, there is a certain risk of it ending up in the wrong place and giving rise to leukemia. This is why gene therapy is only used for serious diseases for which there is no good treatment,
- ethic problems
- Gene therapy is short-lived
In order to achieve long-term benefits of gene therapy, the genes that are inserted into the patient must remain functional after treatment. However, most types of cells that receive new genes during gene therapy eventually die and are replaced by new ones. Currently, researchers have not discovered an effective way to integrate therapeutic genes into a patient's genome. As a result, patients need to undergo multiple treatments of gene therapy in order for it to be effective.
TESTING 1,2,3
Hello! I was thinking that having a blog will be easier for us to post our ideas, instead of emailing things around! Currently only me and Aisyah are the blog authors, cause I don't know Becks gmail address!!
Okay anyway, I shall post some research things up later on!
hope this works. fingers crossed!
wanqi
Okay anyway, I shall post some research things up later on!
hope this works. fingers crossed!
wanqi
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