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Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

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Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#21
post #19

Earlier quoted context omitted.

You have a pretty rosy view of professors. I don't think being a professor means you've won any academic games. You don't get to take sabbaticals when you want. Professors have a lot of people to answer to- granting agencies, deans, students, and more.

>>Professors have a lot of people to answer to Nope. The agencies you mentioned effect their ability to grow, their formal employer is the University. If their research goes to hell they can teach a class and make the same amount of money. Sometimes their students are completely free (TAs). Having seen professors give projects they know dont work and falsify work under vagious euphemisms, they are the problem with ac…

I used to be a postdoc at berkeley and I've seen older professors exited from the university to make room for younger ones- even though they were extremely good teachers.

You can't make the same amount of money just teaching classes- professors have to bring in money to pay their summer salaries.

Note, also, that the general trend in universities has been to move away from full professorships- while the tenure program is great, it reduces the freedom of the administration when it comes to dealing with low performers (I'm not making a value judgement, just an observation).

It don't know what it means for biology to be less mathematically difficult (I'm a biophysicist by training and I assure you the math is pretty hard, especially when you get into grad-level quantum chemistry).

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#22
post #9

Excuse my naive questions, but this left me very curious. 1) Have we reached a phase where the difficult part is finding the genes responsible for X? With the delivery being now boilerplate code (CRISPR?). 2) What exactly are the limitations of gene therapy? 3) How does aging fit into the picture? For example, if grey hairs are the result of cell aging/damaging, will we be able to reverse/restore it?

1) for the most dangerous of diseases, we tend to know their mechanism (gene(s) responsible), precisely because if they're dangerous then they are interesting genes and have been studied in the lab already. That's not to say we know everything - but that information about genes is currently not limiting (for the field).

2) But what you can do in a close environment of the lab with clean rooms, infinite cells, lots of time, infinite do-overs, 30% success rates, and single-cell type environments you can't do in a live body. We've taken apart enough 'cars' to know how the engine mostly works - which are the ignorable parts and which are critical. We've even practiced remaking certain parts, even being creative about making better parts (synthetic bio) to upgrade the engine - however - doing all of the above on a running car that's going 60mph is a whole different story. Currently, the largest challenge is delivering your genetic payload to exactly, and only exactly where you want it. Nowhere more, nowhere less, nowhere wrong, and just right. That delivery is key (and part of why Cas9/crispr is a big deal, it solves part of the problem (where in a genome)). But even if we can target where in a genome, we still need to target where in an organism, and where not in an organism. Delivery is the current limitation. You are made of 3 billion base pairs of information duplicated between a few trillion cells; A 0.00001% mis-delivery rate of this absolutely stunning, perfectly designed new gene/part/function is likely unacceptable.

3) Aging is a lot of things depending on who you ask. Generally mammalian cells are designed to stop growing - otherwise you'd be a thousand-pound sphere of goop at this point rather than a well-defined human with shape. This is a good thing. But it means there are built-in limits on how many times a cell intended to divide. Imagine a book with blank pages up front and in back, where the copy machine can't copy the covers, so it always skips the first and last page. Human cells have about 60 blank pages before they start eating into the text (genetic code) at which point they hit their last life and just try to never die (senesce). There are things we can do to add more pages with genetic therapy, but you got to be careful you don't cause rampant growth (cancer). SO aging is tricky, and a huge frontier that we know very very little about (hard to do experiments where your mean time to finish the assay is 60 years). But there's nothing inherently intractable about the concept that gene-therapies shouldn't be able to affect.

Small molecules ('drugs' as you think of them), are great at killing invaders - things that are distinctly non-human (bacteria/viruses/fungi/etc.). They are not good at affecting 'disorders', cancers or other issues where your own body is over/under/mis-reacting. For that you need to change/alter/upgrade the body's own toolkit. This ability is what synthetic biology promises - access to that toolkit.

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#23
post #2

I work in gene therapy; expect a lot of headlines like this in the coming years. We're just getting started with solving the easy-to-fix problems and disorders right now, but they're falling quickly. Soon it'll be possible to seriously talk about improving working functionality via gene therapy-- I'd expect simple musculoskeletal augmentation first.

I made an 1 day e-mail, so if you can send me your e-mail I will write to you all my questions I have. The mail is navi54@yopmail.com .

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#24
post #10
post #8

Earlier quoted context omitted.

I didn't go the academic science route, but I can maybe tell you a bit about some people who did/are currently. I'll start at the very end, then work backwards: The professors: These guys lived the dream from start to finish. They now have multiple postdocs working to answer the scientific questions that interest them. The professors are responsible for fundraising and presenting the research, and are almost never in…

Thanks for your answer. I have a lot more to ask, but I did not find any email on your profile when I clicked on your name. This site has not PM system, my mistake.

Just email me at cryosin@gmail.com and (if you would) please post a reply to one of my comments here saying that you emailed me, since I don't check that account often.

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#25
post #13
post #8

Earlier quoted context omitted.

I didn't go the academic science route, but I can maybe tell you a bit about some people who did/are currently. I'll start at the very end, then work backwards: The professors: These guys lived the dream from start to finish. They now have multiple postdocs working to answer the scientific questions that interest them. The professors are responsible for fundraising and presenting the research, and are almost never in…

Good luck man, i wonder what the industry is doing differently than academia to speed up the process. Also what is the course of the most passionate people, academia or otherwise?

So far I'd say that academia has more passionate people whereas industry has more effective people.

I'd also say that the industry people seem to think that the passion of academics is related to their narrowmindedness/tunnel vision/stubbornness/navel gazing.

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#26
post #21

Earlier quoted context omitted.

>>Professors have a lot of people to answer to Nope. The agencies you mentioned effect their ability to grow, their formal employer is the University. If their research goes to hell they can teach a class and make the same amount of money. Sometimes their students are completely free (TAs). Having seen professors give projects they know dont work and falsify work under vagious euphemisms, they are the problem with ac…

I used to be a postdoc at berkeley and I've seen older professors exited from the university to make room for younger ones- even though they were extremely good teachers. You can't make the same amount of money just teaching classes- professors have to bring in money to pay their summer salaries. Note, also, that the general trend in universities has been to move away from full professorships- while the tenure progra…

>>It don't know what it means for biology to be less mathematically difficult

It means that wet lab biology can be learned after a few years of work, making professors less valuable as a source of knowledge.

>>grad-level quantum chemistry I would be suprised if the guys running the elisa have more than second semester calculus. Certainly not a requirement for undergrads at our school.

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#27
post #21

Earlier quoted context omitted.

I used to be a postdoc at berkeley and I've seen older professors exited from the university to make room for younger ones- even though they were extremely good teachers. You can't make the same amount of money just teaching classes- professors have to bring in money to pay their summer salaries. Note, also, that the general trend in universities has been to move away from full professorships- while the tenure progra…

>>It don't know what it means for biology to be less mathematically difficult It means that wet lab biology can be learned after a few years of work, making professors less valuable as a source of knowledge. >>grad-level quantum chemistry I would be suprised if the guys running the elisa have more than second semester calculus. Certainly not a requirement for undergrads at our school.

You're talking about technical work, not biology. The same applies to any number of other domains. Sounds to me like you just have a chip on your shoulder.

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#28
post #27

Earlier quoted context omitted.

>>It don't know what it means for biology to be less mathematically difficult It means that wet lab biology can be learned after a few years of work, making professors less valuable as a source of knowledge. >>grad-level quantum chemistry I would be suprised if the guys running the elisa have more than second semester calculus. Certainly not a requirement for undergrads at our school.

You're talking about technical work, not biology. The same applies to any number of other domains. Sounds to me like you just have a chip on your shoulder.

>>You're talking about technical work, not biology.

Well, people get PhD for it. Tell them its not biology :-)

>>just have a chip on your shoulder

Yeah, I blame the academic system

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#29
post #27

Earlier quoted context omitted.

You're talking about technical work, not biology. The same applies to any number of other domains. Sounds to me like you just have a chip on your shoulder.

>>You're talking about technical work, not biology. Well, people get PhD for it. Tell them its not biology :-) >>just have a chip on your shoulder Yeah, I blame the academic system

Nobody gets a PhD for doing lab work. People get PhDs for publishing papers and they do lab work because there isn't enough grant money or capable techs for doing really hard experiments.
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