After reading another article regarding the results of a gene therapy clinical trial, it is abundantly clear that this type of research needs to continue. There is a lot we have to learn and in the case of Layla who was unresponsive to other treatments, we should quickly try to make therapies like these open to everyone as soon as possible. Listen to my podcast to learn about the new therapy that helped a baby girl named Layla.
https://soundcloud.com/user-578021309/audio-recording-on-Friday
References:
http://www.nytimes.com/2015/11/06/business/a-novel-cell-therapy-untested-in-humans-saves-baby-with-cancer.html?_r=0
Friday, November 13, 2015
Friday, November 6, 2015
Chapter 8: Final Thoughts
After spending the past few weeks doing research on genetic
engineering and gene therapies it is very clear there is a lot more research to
be done to test the efficacy as well as the possibilities for treatment of
patients. These therapies would be extremely beneficial and if created
correctly, are thought to permanently cure those with certain congenital
diseases. There is some debate however, about whether or not the therapies
would be considered ethical due to their nature. Some research has shown
promising treatments that have used stem cells to help regenerate cells while
the more accepting therapies use viruses as vectors.
I have a very great feeling that this technology will become
very prominent in the healthcare field and how we go about treatment. Ever
since President Obama’s speech on the Precision Medicine Initiative, a lot of
focus has been on how we can individualize treatment to our genomes. As we
become more familiar with genes and which sequences are responsible for
diseases and disorders, we will better be able to determine which method of
therapies and engineering are best. Initially these therapies will be very
expensive, but it is abundantly clear that overtime there will be a price
decrease, mainly due to the fact that a lot of pharmaceutical companies are
hopping on board with clinical trials and research. Individualized healthcare I
feel is long overdue and gene therapy can potentially be a great part of this.
President Obama's Precision Medicine Initiative speech
References:
https://www.youtube.com/watch?v=MKiw7yAqqsU
Wednesday, October 28, 2015
Chapter 7: Gene Therapy may be the Cure for Retinal Dystrophy
An article was published on the New York Times entitled, “Eye
Treatment Closes In on Being First Gene Therapy Approved in U.S.” by Andrew
Pollack. In the article, the journalist discusses how gene therapy could be an
opportunity much tangible for those seeking treatment in a rare type of genetic
disease that causes blindness. The research that was described seemed really
promising claiming that not only did it stop the progression of the blindness
but in some cases had restored vision in some patients. From a public’s
perspective, this is extremely exciting news. However, this article appears to
be missing some information. Looking at the journalist himself, Andrew Pollack
has his Bachelor’s and Master’s degrees in civil and environmental engineering
so I feel as though he may have not have been able to address the proper
questions he should have in terms of this new biological technology. It appears
as though the side effects of this treatment were only noted as being minor,
however, in every article that I have read thus far about gene therapies the
list of side effects has been extremely long.
Thursday, October 22, 2015
Chapter 6: Some General FAQs about Gene Therapies
Q: If I want a child with no “bad” genes, how much would
that cost to find if the embryo has them or not?
A: Approximately $3,500 in the United States and twice that
in Britain. This process includes testing the embryos for genetic defects as
well as in vitro fertilization.
Q: How likely is it I can choose my child’s sex?
A: Apparently very likely. According to John Hopkins
University, research has shown that as of 2006 65% of clinics have this
ability. Now that it is 2015, it is likely to be higher.
Q: How are genes delivered in gene therapy?
A: Genes are actually injected into viral vectors which then
“infect” you cells and implant the correct gene into your cell’s DNA.
Q: Can these viruses make me sick?
A: Not at all. The viruses used thus far in clinical trials
are under very strict guidelines to insure purity and integrity of the DNA that
is being transported into your cells.
Q: What is the difference between gene therapy and cell
therapy?
A: Gene therapy is the act of injecting a virus into the
blood stream which then injects the correct form of the gene into your cells.
Cell therapy is the act of injecting actual cells into a particular area of the
body that have relevant function.
Q: What kind of diseases can gene therapy treat?
A: Gene therapy is thought to be a great candidate for
fixing any genetic defect in our DNA. From Cystic Fibrosis to possible Cancer
treatment usage, there are a lot of options for what may come of this
technology.
Q: Why would we want to use Stem cells for cell therapy?
A: Stem cells are able to differentiate into any cell in the
body and have a lot of regenerative qualities that could be sustained
throughout the lifetime of a patient. Stems cells also would eliminate the
concern of the immune system rejection, where the immune cells basically attack
thinking that the foreign material is bad. With the use of Stem cells, it would
already be the cells of the patient and the body would adapt perfectly. References:
http://image.slidesharecdn.com/genetherapy-131009190236-phpapp02/95/gene-therapy-3-638.jpg?cb=1381345440
Wednesday, October 14, 2015
Chapter 5: Are Designer Babies Just a Fad?
The idea of Designer Babies is more prominent now than ever.
Personally, I find this field extremely interesting and so I did some research
from the perspective of a parent that would be in interested in such technology.
Just searching the term “Designer Babies” on the internet, the first link that
comes up is a link to “The Fertility Institutes”. When you click on the link,
the first thing that pops up on the screen is a representative live chat where
they can instantly reply to any questions you have. It appears as though the
first factor someone that is having a Designer Baby would be interested in is
gender, not the possibility of removing a genetic disease.
Screenshot of the web page http://www.fertility-docs.com
Honestly, I found this sign alarming. The term Designer Babies
has been sensationalized, the idea of gender selection specifically. On the
front page of the website, the institute shares a link to an article about how
Kanye West and Kim Kardashian have already taken part in gender selection for
their second child who is on the way. There is also a link to a video of a “Good
Morning America” clip where Dr. Steinberg, the found of The Fertility Institutes
has a segment on the show called “Family Balancing”. The institute has also
been recognized on the networks ABC, NBC, CNN, Fox, and Reuters as among the
worldwide leaders in gender selection.
Interestingly enough, there is little to no mention of the
cost of the technology and nearly no mention of the mutational risks that could
happen due to the manual moving of the sex chromosomes by scientists. From my perspective
it is disturbing that looking at just this one website page people would be
willing to be a part of this technology so blindly and by looking at one simple pamphlet.
References:
Thursday, October 8, 2015
Chapter 4: The Possiblities for Gene Therapy in Patients with Haemophilia
A review written by The Cochrane Collaboration entitled, “Gene
Therapy for Hemophilia” has some telling information in the pros and cons of
what gene therapy has to offer the medical community as well as how a clinical
trial should be set up. This review discusses how promising gene therapy is for
people who are greatly affected by genetic diseases and how this could be used
for things other than just hemophilia in the future. Hemophilia is a X-linked
recessive gene where clotting factors are unable to function and excessive
bleeding occurs in patients.
Something I found interesting was that the clinical trial
that would be done for haemophilia would have a lot of cons. Unfortunately,
because the gene therapy treatment is not being mass produced it is extremely
expensive. In the case of those affected by haemophilia, the therapy would have
to be administered every two to three days because of the short half-live of
clotting factors in the blood. The price for treatment was not listed, but it
can be assumed that at that rate, it would be extremely hard to keep purchasing
injections for therapy. There were some interesting points brought up about the
side effects of this therapy such as the fact that some patients’ might not
adapt well because the treatment introduces new genes into a cell’s DNA which can
cause some insert mutations and could leave the patient worse off perhaps
giving them another genetic defect.
In contrast, there are an immense amount of opportunities
that gene therapy could offer for those affected by genetic disorders. It is
believed that if this therapy is successful many patients would have a lifelong
cure and mortality rates would greatly decrease. Not only the patients would
benefit but the researchers and medical physicians would as well. By gaining
knowledge about how gene therapy is successful could help broaden the types of
diseases that are treated.
References:
Sharma A, Easow
Mathew M, Sriganesh V, Neely JA, Kalipatnapu S. Gene therapy for haemophilia. Cochrane
Database of Systematic Reviews 2014, Issue 11. Art. No.: CD010822.
DOI: 10.1002/14651858.CD010822.pub2.
DOI: 10.1002/14651858.CD010822.pub2.
Thursday, October 1, 2015
Chapter 3: Biomedical and Pharmaceutical Companies are Hopping Onboard
After
reading the article written by the Boston
Globe’s Robert Langreth titled, “Money flows again to gene-therapy drugs
investors once shunned”, it is abundantly clear that gene-therapies are in our
near future for treatment. Although there are still a lot of research that is
being done to make sure that the therapies are safe, there are a lot of
biomedical and pharmaceutical companies that are hopping on board in terms of
funding.
This
treatment approach has always appeared to have a lot of promising points even
after some patients suffered fatal side effects in the 1990’s and the early
2000’s. Thanks for financing that enabled more research to be done in the field
there have been some extremely promising clinical trials that show new delivery
methods that are much safer for patients. Most recent clinical trial successes
have been for patients affected by hemophilia, eye disease, and cancer. The
funding that is being done by biomedical and pharmaceutical companies are
appearing so promising that there are actual companies that are opening stock
specifically for gene therapy technology such as the therapeutic company,
Regenxbio.
From a
consumer perspective, with all of these companies that are being involved it
really begs the question of how much will this therapy cost and how will
insurance companies be a part of it, if at all? The article from the Boston
Globe does not touch upon how it will be available to patients and I feel as
though that is a huge point that should have been mentioned.
References:
http://www.regenxbio.com/
https://www.bostonglobe.com/business/2015/05/20/money-flows-aain-gene-therapy-drugs-investors-once-shunned/8SfciTEqF032uDRS4fhpxN/story.html
Tuesday, September 22, 2015
Chapter 2: Gene Therapy and Cystic Fibrosis
What is Cystic Fibrosis?
Cystic Fibrosis (CF) is a hereditary disorder that affects
the cells of the lungs, pancreas, and small intestines. This disease causes
abnormal amounts of mucus to be secreted from these regions and usually results
in coughing, poor digestion, and extremely higher risks of infection. If a
patient goes untreated they will most likely develop lung infections that are
fatal and more likely than not will not live past their late 20’s. Cells that
are within the lung’s lining produce a lot more mucus because of a mutation
found on chromosome 7 that is rightfully called the Cystic Fibrosis
Transmembrane Regulator (CFTR) gene. This mutation is a deletion of an amino
acid that completely changes the overall function of the gene.
Photo courtesy of the NIH
How can Gene Therapy help?
Cystic Fibrosis appears to be an excellent candidate for
gene therapy because adding the gene that was deleted would most definitely fix
the mucus production of the cells. So how do we do it? The first step is to
find a vector to carry the normal gene into the body. By injecting the gene
into a virus, the virus will be able to “infect” the cells with the correct
gene and help control the mucus production.
Let’s do it!
This idea of gene therapy sounds so easy to accomplish,
however, like anything else, it requires a lot of funding. Currently, there are
patients and scientists undergoing clinical trials for this research but it is
going much slower than anticipated. The idea of gene therapy is looking
extremely promising and seems like a beneficial outcome of genetic engineering.
References:
Wednesday, September 16, 2015
Chapter 1: An Introduction
Ever since the Human Genome Project, science and researchers
have been increasingly more and more fascinated by genetics and the DNA that
makes us who we are. Genes influence traits and behaviors as well as health and
disease and although the science community has learned a lot, there is so much
left for the science community to learn about what makes people who they are.
The Human Genome Project discovered that there are approximately 20,500 human
genes. This completed sequence allows us to identify the locations of genes and
the overall structure and organization of our genome. This knowledge has set a
basis for what research we are doing on genes in general.
The Cons to this Technology
When most people think about
Genetic Engineering, the idea of “Designer Babies” always appears to come up as
a topic. From a parent’s perspective, it would seem incredible if we were able
to remove a genetically linked disease from our child’s embryo. Unfortunately,
this leads to the question of where does this genetic-tweaking end? What are
the moral/ethical implications of making sure that our child visually looks
adequate according to society? It also questions the child’s lack of autonomy
for these decisions because they are decided at such a young age. Many people
argue that this idea of “Designer Babies” is just the beginning stages of
eugenics and we should stop research.
Photo courtesy of University of
London Forum on Genetic Engineering
The Pros to this Technology
Although there would seem to be a lot of downfalls for this technology there are actually a lot of possible positive outcomes of such research. One thing in particular is gene therapy. Gene therapy is a technique that is being used as a treatment as opposed to drugs or medication. By using this therapy doctors and scientists are allowed to treat a disorder by way of inserting a gene into a patient’s cells. As of right now, the treatment is still under study but appears to show a lot of promise in terms of treating inherited disorders, types of cancers and some viral infections.
Although there is still so much to learn in this field of Human Genetic Engineering, there appear to be a lot of promising research that will hopefully benefit science and healthcare treatments in the long run. Overall, the subject needs a lot more research done and we are already making a decent amount of progress.
References:
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