md:genetherapy
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md:genetherapy [2021/06/22 01:38] admin created |
md:genetherapy [2021/06/22 01:39] (current) admin |
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| - | - last 5 years - a renaissance in gene and cell therapy --> first approved therapies after decades of efforts | + | - last 5 years - a **renaissance** in gene and cell therapy --> first approved therapies after decades of efforts |
| - approved | - approved | ||
| - oligonucleotide, | - oligonucleotide, | ||
| Line 15: | Line 15: | ||
| - authors suggest a time-line | - authors suggest a time-line | ||
| - outstanding issues: | - outstanding issues: | ||
| - | - immune response: gene delivery vectors, products of foreign transgenes -- AAV based; how about others? 50% excluded | + | - **immune response**: gene delivery vectors, products of foreign transgenes -- AAV based; how about others? 50% excluded |
| - new developments: | - new developments: | ||
| - better DNA editing and repair | - better DNA editing and repair | ||
| - RNA editing | - RNA editing | ||
| - stil many unknowns: 6K of 20K human genes function unknown | - stil many unknowns: 6K of 20K human genes function unknown | ||
| - | - epigenetic regulator function not well known but clearly important | + | - **epigenetic** regulator function not well known but clearly important |
| - | - CRISPR (CTX001) targets distal gene regulatory element rather than underlying genetic mutation | + | - CRISPR (CTX001) targets distal gene regulatory element rather than underlying genetic mutation |
| - | - limit of regulatory models for gene therapy treatment | + | - limit of **regulatory models** for gene therapy treatment |
| > Gene therapy is arguably the most exciting area of biotechnology at this moment - both due to recent progress and because of the possibilities on the horizon. Unprecedented levels of control over nucleic acid delivery, modulation of the immune system, and precise manipulation of the human genome – technologies not imaginable ten years ago – will certainly unlock new areas of medicine over the next ten years. At the same time, this nascent glimpse of a new world of technical capabilities has inspired whole new areas of research, such as synthetic biology, cell reprogramming, | > Gene therapy is arguably the most exciting area of biotechnology at this moment - both due to recent progress and because of the possibilities on the horizon. Unprecedented levels of control over nucleic acid delivery, modulation of the immune system, and precise manipulation of the human genome – technologies not imaginable ten years ago – will certainly unlock new areas of medicine over the next ten years. At the same time, this nascent glimpse of a new world of technical capabilities has inspired whole new areas of research, such as synthetic biology, cell reprogramming, | ||
md/genetherapy.1624325887.txt.gz · Last modified: 2021/06/22 01:38 by admin
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