Just wondering what you guys think are some of the top gene therapy research universities. Any and all feedback welcome! Thanks!
Top Gene Therapy Research
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penn - although he hasn't always been successful (and has had extremely public fiascos), jim wilson is undoubtedly one of the fathers of gene therapy and vaccine development
quit posting just to post - i just said that (albeit in a less straightforward manner) - but yes, that is the same person
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pfft, i asked because i was curious. i took a class on muscular dystrophies last year and the topic of AAV vectors came up - i didnt know who was behind it, or whats the background of the research. all i heard was bits and pieces from the prof
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Just a friendly warning from an 8th year MSTP who was also interested in gene therapy....
DO NOT DO A PH.D. IN GENE THERAPY.
Your time would be better served banging your head against a wall.
Wow, that's a strong statement. 8 years of banging your head against a wall would, I think, end with not much of a head to bang. From somebody else potentially interested in the field, could you elaborate?
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Not much more to know other than:
1) no matter how good your work is, it will not be relevant clinically with all the bad press GT gets and you will never likely see clinical trials because getting IRB approval is basically impossible
2) for the above reasons and many others, it is virtually impossible to get your work published
3) LONG commitment developing animals for therapy, with little chance of success
4) NOT hypothesis driven work; if you don't get the desired result you can't publish AND you've learned nothing- back to square one if you want a Ph.D.
I've seen several people do GT at my institution in HH funded labs- they had very long Ph.Ds (6+ years) and plead for mercy to get out. One person I know graduated sans publication, despite working his arse off.
Don't forget that you're in grad school to earn a Ph.D., not to cure cancer. Find a good lab with interesting projects and money (and people you get along with). Get a Ph.D., then work on whatever you want.
For the record I don't think Jim Wilson works directly on gene therapy any more... someone from Penn will have to chime in but as I understand it he can only consult on such projects since... "the incident".
Gotcha, makes sense. Given the vehemence of your advice, I think I'll take it. (though I do think gene therapy is fascinating)
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I have a friend here in my year doing a PhD in gene therapy. I think that person would say similar things... I've seen some very unhappy unhappy e-mails from that person.
BTW, I have no diea what Jim Wilson is up to these days. I'm more on the engineering side of things, even if I'm not an engineer.
I agree. I've been in the field for about 3 years as an undergrad and in that 3 years not a single grad student has made ANY progress...are they 3 years closer to finishing their PhD? Hellz no!
Thus I am getting out of the field but taking the skills and techniques I learned...
Well he certainly didn't talk about gene therapy in the classic sense although his lecture (during the graduate school part of the interview trip) did talk about using AAV to develop a vaccine for Ebola...
Basically his lab is creating a transgenic adeno-associated virus with 1 or 2 Ebola genes 'spliced' in...
Not sure what else his lab is working on...I heard it's a mega-lab (50+ people)
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Dr. Katherine High (at University of PA) is one of the leaders in GT. She is very close, I think, to developing an FDA approved GT for treating patients with hemophilia B.
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As a person who has just graduated from undergrad and worked in a gene therapy lab, I can say that what you are saying is wrong and I am a perfect example of why you are wrong. I work studying vector therapies for malignant brain tumors. Answering in the order they were given:
1) There are several kinds of gene therapy (adenovirus, cells, immuno-gene therapy) that have been tested in clinical trials around the world, recently and more continue to be evaluated for entrance into phase 1 clinical trials. I know this because we're working with the FDA right now to have a vector approved. This leads me to the next question.
2) I know this because I just had a review accepted about vector therapy clinical trials and recent developments in vector therapy for brain tumors. Also, I've been published in six other peer-reviewed research studies either as 1st author, second, or third. You can, in fact, get published.
3)Developing animal models is tough, but success is possible. Your outlook is bleak.
4)How can you say that any research study is not hypothesis driven ? In GT, the hypothesis is that your genetic maneuvre will be better than others tested for reasons 1, 2, 3, ...n. This doesn't constitute a hypothesis? Also, I've learned so much in my lab about, genetics, virology, immunity, imaging, biochemistry, transduction, etc. It's been great. Perhaps these two outlooks can represent the both really positives and really negatives of gene therapy research. It's a tough road, but you have to go about it the right way. Gene therapy for cancer research is more viable, especially for the malignancies with low survival rates. I am lucky enough to be part of lab that has just gotten really fiscally solvent, so we can do a lot and sometimes this factors in on the rate of publishing/impact of publication. It's not impossible. I think GT is pretty complex and still in infant phase of development, but it's a pretty sick area to study nonetheless. About institutions:
University of Alabama (UAB) has a division of gene therapy that works in concert with many depts. like pathology, oncology, surgery, etc.
Ohio State university has new neurosurgery chief who is big investigator of gene therapy for brain tumors
I think Duke is doing some clinical trials too.
This is all i can remember now.
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Startrack,
First off, congratulations on your success. Having several publications, especially at your stage in your career, is very impressive and I have no doubt you will be successful.
Second, let me clarify some of my statements. Having success in GT is possible, as you have seen. However, from what I have seen, it is a much more difficult and a less fruitful road. I don't know where you've worked or in what journals you've published. I am at one of the premier human genetics institutions in the US and got my Ph.D. in the genetics department. I have seen many people attempt such projects. Very few have had success, and the ones that do take significantly longer to graduate than their peers. As I said, only one person graduated without a publication, and I do think that after graduation he was able to publish his work.
Third, designing vectors and gene therapy projects are generally NOT hypothesis driven. Showing that one treatment modality or vector works better than another does not really constitute a viable Ph.D.-caliber hypothesis. Such a hypothesis would suggest why one thing happened and not another; not simply that it works (this would just be an observation). As you will learn in graduate school, a hypothesis must be falsifiable. You really need to fulfill both of these requirements. Why am I babbling about this? If you spend three years creating a mouse and a vector to test, and in the end your gene therapy of gene Y is fruitless, then you have learned nothing and have to start over. If, however, you spent that time studying how gene X interacts with protein Y to activate gene Z, but in the end gene Z is not activated, you will have learned a lot about the mechanisms of proteins Y and genes X and Z, and can readily formulate a new hypothesis. The point is, it's not a "yes/no" answer, and you will learn something, publish and graduate in a timely fashion.
Of course, finally, I will re-iterate that publishing in GT is more difficult in other fields. There may be exceptions like super deadly cancers or whatever, but in general what I said above is true. You can always work on vectors alone in an engineering lab, but this isn't really GT and is generally called virology.
Cheers
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First of all, thanks for the crash course in intro to genetics, gb. I forgot that it takes several years of grad-level research experience at a premier U.S. genetics institution to understand gene regulation. I was just providing a counter to your earlier post in this thread where you listed all the reasons not to do gene therapy.
As I'm sure you learned getting your PhD in genetics, you can't make assertions in science with absolutes like 'impossibe' and 'you've learned nothing'. I wouldn't want one person to not go into a field because of the negative second-hand experience of some person they don't even know.
And I still can't believe that you're saying GT isn't hypothesis driven. You must be REALLY jaded with research. Thanks for clarifying.
As I'm sure you learned getting your PhD in genetics, you can't make assertions in science with absolutes like 'impossibe' and 'you've learned nothing'. I wouldn't want one person to not go into a field because of the negative second-hand experience of some person they don't even know.
And I still can't believe that you're saying GT isn't hypothesis driven. You must be REALLY jaded with research. Thanks for clarifying.
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ok, first off, as a person who is not extremely interested in gene therapy and what it entails, I would say, as an outsider to the subject, that you are misinterpreting what gb is saying. I don't think gb was talking down to you when talking about looking at the regulatory components of a pathway, merely the positives of taking that route to study a gene rather than gene therapy..second while you seem to be a very accomplished undergrad and have done great things already (again as gb states) you need to remember that you still are an undergrad, gb is almost done with his/her MD/PhD, and subsequently has almost 10 years of academic experience on you..now that could mean everything or nothing but I would think that gb's comments are worth more than second hand experience of someone we hardly know (i mean it is a public forum how well can you actually know someone that contributes ?)
anyways I'm sure if you have some sarcastic remarks for me I probably do deserve them but while gb may have come on a little too strong by saying "don't do gene therapy" I think that gb's opinion should be taken with more than grain of salt? Ask yourself this, would you feel as strongly about gene therapy if you had not succeeded at all as an UG??
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I do, indeed.
Taxpayers of America, I'd like for you to meet our future physician scientists ! (havoc and rioting ensues)
I apologize to all the lovely regular forumites for making the aura of this forum even muddier BUT....
after reading ALL of your previous 37 posts or so, I ask myself (and subsequently you as well) if you are posting in this forum because you're sincerely interested in life as a physician-scientist or because you like to make trouble.
So far every single one of your posts has been narrow-minded and anecdotal at best, but most have been immature and rude...
Oh and before increasing the sarcasm in your responses, try adding something productive to this forum besides one little post - I may not be the biggest fan of gbwillner (always making me feel stupid) BUT he/she has been one of our invaluable resources...
basically - if you can't say anything nice or productive then go back to pre-allo
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Demoralizing...But it makes sense. Does the same apply to immunotherapy?
My advice for a PhD thesis project is always against therapy-driven projects that are goal oriented.
Let me explain with a colorful analogy... to baseball. Making great advances in GT and immunotherapy is like hitting a home run. studying genes and proteins sounds much less sexy, and is probably like getting a single. However, your goal is to score (get a Ph.D.) and to do that you need to get on base. Home run hitters are flashy, but they are slow and have low batting averages. Guys who hit for average score more runs and are quick. You can't always swing for the fences- you will strike out far more often than not. OK, I see this is getting thin.
Let me explain some reasons why this is not the best Ph.D. project. First, when you walk into a therapy project, chances are it's been in the pipeline for some time if you're actually going to come to some trial. That means that you are not the primary thinker in the project and that will rob you of great experience. If it's much earlier than that, then you will need a LONG time to get to that point- either developing mice or vectors or both.
Secondly, these projects are not really science- and what I mean by that is that they do not really use the scientific method to any great extent (wherein you try to understand why one thing is while another is not). They are GOAL oriented- you want to find a specific outcome (curing AIDS or whatever) and you speculate that your treatment modality will achieve that goal or not, or some therapy is BETTER than some other, existing therapy. The results are either: you succeed or you do not. This is more like engineering, or applied science. Same as for projects that create vectors for a specific purposes (can I create a vector that targets cell type Z yes or no?). This can make great advances in our lives, but they make poor thesis projects. If the answer is yes, maybe you'll get a Ph.D., but members of your committee will complain that you didn't really work that hard and didn't follow the scientific method. But these projects can be difficult to publish, or more likely, not work at all. If this is so, you have learned little other than your treatment was not good. If, on the other hand, your project involves studying the mechanisms of how the gene you're targeting is affected by certain transcription factors that are known to activate/silence your gene/protein/whatever, you will ultimately succeed, even if your hypothesis is wrong. You will learn enough about those interactions through your experiments to make better hypotheses to test. It's not all-or-nothing. You may find out that you were totally wrong initially, but find some other important aspect of that gene's function that in the end is far more interesting.
Now keep in mind that in other fields this is perfectly acceptable- notably engineering. Also keep in mind that many people do these kinds of projects and CAN succeed, but in my experience they are the ones that are more bitter and have longer PhDs or quit. Also, I think it is great to work on such projects- just don't make them your thesis work. Then if they fail, no biggie. Go back to your primary work. It's good to take some risk, just don't put all your eggs in one high-risk project.
But in your specific question, I do think immunotherapy is better than GT for the time being, and probably sexier. but, as a thesis project, it may be better to focus on immune responses to specific surface antigens and how they could be applied for therapy than to actually attempt such a trial. Save that for when you have your own lab and ample funding.
As for Star Track's comments they are hardly worth a response. I wish that when I was at your stage I had access to this type of a forum where I could get the advice of people with much more experience than I and was willing to share it. I may be brash at times, but I definitely have your guys success in mind with my comments. Good luck to you guys.
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For the most part, I agree with gbwillner's above post. Generating a vector that 'works' is very difficult and it has taken many years of hard work and collaboration with other gene therapy units for our group to finally come up with a good vector. Going into GT therapy in order to create that 'ideal vector' would be very painstaking because people have been trying to do that - depending on the particular field which GT is applied to - for decades. Personally, I think that I would more agree with what gbwillner is saying with regard to immunotherapy; ultimately it really depends on your approach to the field. But given your approach, your research can also have relavance to gene therapy. What I was interested in doing for a PhD would be investigating particular patterns/interactions/biochemistry of an important gene of a GT vector. Ultimately, this can still be accomplished within the scope of GT because your findings could lead to the identification of a better vector, but can also qualify for a study in another area such as genetics/biochemistry/molecular virology because your model (the vector) serves as a good system for analyzing gene expression/regulation/biochemistry/something that is relevant to your interest of study. In this way, your investigation will still lead you to a unique discovery and a degree, whether or not you create that ideal vector. You stilll will have shown something important in genetics/biochem but also shed some light on an unknown aspect of GT that many in the field will find important. Also, if you're creative about it, you can find many ways to publish the little bits and pieces of what you have found along your way. Just don't get set in on always publishing in cell, nature, PNAS, science, or some high impact journal like many others. I hope this is helpful to some.
If you don't believe me, maybe you'll find this review helpful:
http://www.ncbi.nlm.nih.gov/pubmed/16705130?ordinalpos=1&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_RVLinkOut
If you don't believe me, maybe you'll find this review helpful:
http://www.ncbi.nlm.nih.gov/pubmed/16705130?ordinalpos=1&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_RVLinkOut
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Also, keep in mind that some GT fields are more difficult than others. Working with things like retrovirus and stuff can prove to be VERY difficult because a lot is still unknown regarding the viral vector itself. Also, be careful with some of the heavy biotech stuff. There is this research associate who works in my lab doing stuff like hybridoma technology. She does things like generate single chain antibodies with enhanced tissue targeting as the end goal. This sort of stuff can be very painful because it includes many steps and therefore many chances for things to go wrong. The way I feel about it, the more you try and tinker with something in nature to get a desired outcome, the more difficult it becomes to get what you want. Try and keep it simple and effective, but be open minded to what you see in your results. This can lead to extra publications
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Thank you both for your input. There' a immunotherapy lab that I'm currently looking into as a potential thesis lab. I like their concept and strategy, and the PI seems nice. They already have a working molecule in phase II trial, and I'm certainly not interested in working with that during my PhD. I f I do end up joining that lab, I think I will focus on uncovering new targets, mechanisms and such.
If this continues I'm closing the thread. Please knock it off.
haha how on earth could I possibly be mean with avatar like that in the thread?!
