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Advanced treatments and its transfer to patients
Published on Jun 14, 202326 Views
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Chapter list
Advanced treatment of genetic disorders and its translation00:00
Scientific expertise & technologies at the Department of Synthetic Biology and Immunology at NIC00:34
Research: gene and cell therapy technologies01:07
2 ERC projects for established biomedical scientists01:56
Revolutionary breakthroughs in the life sciences02:12
Pillars of pharmacological therapies03:22
Therapeutic options for genetic disorders03:44
Gene replacement therapy04:17
CRISPR/Cas-based genome editing05:02
Base editors (cytosine, adenosine)06:21
Prime editors07:16
Refinement of PE at NIC07:45
Mechanisms of action of antisense nucleotides (ASOs)08:08
Approval of ASOs for treatment of neurodevelopmental diseases08:56
Nusinersen Spinal muscular atrophy09:07
Our ASO pipeline for CTNNB110:10
Collaboration between researchers, clinicians and patient organizations10:56
Research group and collaborators11:47
Centre for the technologies of gene and cell therapy12:06
Collaboration between clinicians and patients12:28
Establishment of CTGCT13:24
The Vision14:30
The healthcare landscape15:22
Impact15:54
Organization16:29
Bridging gaps in translation16:57
Workflow and partners contribution17:16
Timeline17:59
The team behind the project18:39
Acknowledgements18:48
The bridge between biomedical research on advanced treatments and its transfer to patients19:03