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While I'm sure some variant of that will occur at some point, many genetic disorders are relatively simple and well understood. Even if we are still learning,
by sfeng 10y ago
While I'm sure some variant of that will occur at some point, many genetic disorders are relatively simple and well understood. Even if we are still learning, there are dozens of disorders we could eliminate if we had robust genetic editing capability with pretty good odds of success.
- badosu 10y ago"... many genetic disorders are relatively simple and well understood." Are they, really? As far as I know (and I am not a specialist, so someone more qualified may correct me), gene transcription and translation is far from well understood. It's as if we don't have access to the source code for the compiler, only the machine code and the syntax. You can draw correlations and infer causality from your changes, but you can't really be sure.
- itchyjunk 10y agoNot an expert either: Even when we know how it happens, we don't know when it happens i.e. when certain chunks of replication is triggered and when it's not. Environmental factor, hormones, diet, mix of them all and much more etc.
- sfeng 10y agoWe understand the compiler pretty well. You can grab a Bio textbook and find incredibly detailed diagrams of how the genetic transcription process works. It is true that we don't understand every factor, but it's likely the most significant ones are the ones we understand best.
- mattkrause 10y agoIt depends. At one end of the spectrum, Sickle Cell Anemia is caused by a single point mutation. The sixth amino acid in the hemoglobin beta chain should be glutamic acid, but it's been replaced by a valine. These have different charges, which is enough to warp the cell's shape and impair its function. At the the other extreme, a lot of different gene variants have been associated with autism. However, these tend not to replicate very well--the genes identified in study A don't show up in study B and vice versa, and there are other possible mechanisms, like copy number variants. CRISPR could potentially fix the former, but the latter is way beyond our current understanding.
- jfarlow 10y agoYep. As was in the news a couple of days ago, sickle cell disease can be effectively 'cured' by installing an updated Hemoglobin protein [1]. (Some) Breast cancer associated genes have pretty well understood issues that can be effectively remedied with gene therapies [2]. And mutations in and around the oncogene P53 have pretty well understood effects - all the way through public policy with regards to HPV vaccination [3]. Adding single proteins and flipping single genetic bits in single cell types in single organs is not the kind of thing that generally causes cascade failures. There are always exceptions, but in general biology is pretty robust to such changes. In fact, those are exactly the kinds of changes that occur from generation to generation. And these are precisely the kinds of systems that have been under deep study for the past four or five decades. [1] https://serotiny.bio/notes/proteins/hbb/ https://serotiny.bio/notes/proteins/hbb/ [2] https://serotiny.bio/notes/proteins/brca1/ https://serotiny.bio/notes/proteins/brca1/ [3] https://serotiny.bio/notes/proteins/p53/ https://serotiny.bio/notes/proteins/p53/
- badosu 10y agoThanks for the insightful responses