Advanced treatments and its transfer to patients

Published on Jun 14, 202323 Views

Kleefstra Syndrome Scientific Conference 2023: “Moving towards the uptake and use of Artificial Intelligence (AI) in research and clinical work”, Ljubljana 2023

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