Center for Advanced Studies (CAS) Research Focus CRISPR/Cas
Center for Advanced Studies (CAS)
6 episodes
6 months ago
Emmanuelle Charpentier and Jennifer Doudna received the Nobel Prize for Chemistry in 2020 for their discovery in 2011 that CRISPR/Cas can be used to cut DNA at specific sites – a discovery that revolutionized research, especially in the fields of medicine and plant breeding. With this method, it is possible to make specific cuts and modifications to DNA with minimal cost and unparalleled efficiency (Genome Editing). Due to its simple handling, CRISPR/Cas-assisted gene editing is a technology that is further developing very quickly. Alongside other improvements, there are now methods for introducing CRISPR tools directly into plant or animal cells – without the detour via a corresponding gene construct. In essence: we are now in a position not only to read genomes, but also essentially to rewrite them at will.
However, this new method and its further development also raises a number of legal, social, ethical and economic questions, for example regarding regulation or the establishment of an international moratorium on all forms of germ line intervention in humans. Plant breeding is concerned with the question of whether genome editing leads to plants being regarded as genetically modified organisms even though their genomes look "nature-identical". At the same time, the extent to which genome editing can contribute to the sustainable production and distribution of safe food remains highly debated. Another aspect is the problem of patents: who is entitled to which patents for the CRISPR Cas9 technology? And of course there is the question of how best to communicate this technology – its potentials and also its dangers – to a broader audience and initiate a constructive dialog between scientists and the general public.
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Emmanuelle Charpentier and Jennifer Doudna received the Nobel Prize for Chemistry in 2020 for their discovery in 2011 that CRISPR/Cas can be used to cut DNA at specific sites – a discovery that revolutionized research, especially in the fields of medicine and plant breeding. With this method, it is possible to make specific cuts and modifications to DNA with minimal cost and unparalleled efficiency (Genome Editing). Due to its simple handling, CRISPR/Cas-assisted gene editing is a technology that is further developing very quickly. Alongside other improvements, there are now methods for introducing CRISPR tools directly into plant or animal cells – without the detour via a corresponding gene construct. In essence: we are now in a position not only to read genomes, but also essentially to rewrite them at will.
However, this new method and its further development also raises a number of legal, social, ethical and economic questions, for example regarding regulation or the establishment of an international moratorium on all forms of germ line intervention in humans. Plant breeding is concerned with the question of whether genome editing leads to plants being regarded as genetically modified organisms even though their genomes look "nature-identical". At the same time, the extent to which genome editing can contribute to the sustainable production and distribution of safe food remains highly debated. Another aspect is the problem of patents: who is entitled to which patents for the CRISPR Cas9 technology? And of course there is the question of how best to communicate this technology – its potentials and also its dangers – to a broader audience and initiate a constructive dialog between scientists and the general public.
Safety and Efficacy of CRISPR/Cas9 based Gene edited Hematopoietic Stem and Progenitor Cells in Hemoglobinopathies
Center for Advanced Studies (CAS) Research Focus CRISPR/Cas
56 minutes 42 seconds
4 years ago
Safety and Efficacy of CRISPR/Cas9 based Gene edited Hematopoietic Stem and Progenitor Cells in Hemoglobinopathies
Sickle cell disease is one of the most prevalent diseases worldwide. Even optimal conventional care has demonstrated inferiority compared to curative options such as hematopoietic stem cell transplantation. Gene editing promises an elegant alternative cure to all patients lacking a matched donor, potentially avoiding many of the transplantrelated complications. | Selim Corbacioglu is Professor for Pediatric and Adolescent Medicine as well as Director of the department for Pediatric Hematology, Oncology and Hematopoietic Stem-Cell Transplantation at the University Hospital Regensburg. 2019, he was able to succesfully treat a beta-thalassemia-patient by means of the CRISPR/Cas9 gene therapy for the first time.
Center for Advanced Studies (CAS) Research Focus CRISPR/Cas
Emmanuelle Charpentier and Jennifer Doudna received the Nobel Prize for Chemistry in 2020 for their discovery in 2011 that CRISPR/Cas can be used to cut DNA at specific sites – a discovery that revolutionized research, especially in the fields of medicine and plant breeding. With this method, it is possible to make specific cuts and modifications to DNA with minimal cost and unparalleled efficiency (Genome Editing). Due to its simple handling, CRISPR/Cas-assisted gene editing is a technology that is further developing very quickly. Alongside other improvements, there are now methods for introducing CRISPR tools directly into plant or animal cells – without the detour via a corresponding gene construct. In essence: we are now in a position not only to read genomes, but also essentially to rewrite them at will.
However, this new method and its further development also raises a number of legal, social, ethical and economic questions, for example regarding regulation or the establishment of an international moratorium on all forms of germ line intervention in humans. Plant breeding is concerned with the question of whether genome editing leads to plants being regarded as genetically modified organisms even though their genomes look "nature-identical". At the same time, the extent to which genome editing can contribute to the sustainable production and distribution of safe food remains highly debated. Another aspect is the problem of patents: who is entitled to which patents for the CRISPR Cas9 technology? And of course there is the question of how best to communicate this technology – its potentials and also its dangers – to a broader audience and initiate a constructive dialog between scientists and the general public.
