CTGCT
@ctgct.bsky.social
New center at National Institute of Chemistry in Slovenia focused on translational research of gene and cell therapies for #RareDisease and #Cancer, funded by the EU, the Slovenian Ministry of Higher Education, Science and Innovation, and UKRI.
This led her team to ask a transformative question: could this powerful inflammatory potential be repurposed to treat cancer?
To find out, check the recording of the webinar
ctgct.si/en/objave/ha...
#CTGCT #ScienceTalks
To find out, check the recording of the webinar
ctgct.si/en/objave/ha...
#CTGCT #ScienceTalks
A Nobel-Worthy Topic: Harnessing Inflammation to Fight Cancer - CTGCT
On October 6th, the very day the 2025 Nobel Prize in Physiology or Medicine was awarded for groundbreaking work in T-cell based cancer immunotherapy, our CTGCT community had the pleasure of hosting a ...
ctgct.si
October 10, 2025 at 10:38 AM
This led her team to ask a transformative question: could this powerful inflammatory potential be repurposed to treat cancer?
To find out, check the recording of the webinar
ctgct.si/en/objave/ha...
#CTGCT #ScienceTalks
To find out, check the recording of the webinar
ctgct.si/en/objave/ha...
#CTGCT #ScienceTalks
In her talk, Dr. Iva Hafner Bratkovič introduced the inflammasome, a part of our innate immune system that acts as a “potent inflammatory machine”. While these molecular complexes are essential for protecting us from pathogens, their dysregulation can contribute to a variety of diseases.
October 10, 2025 at 10:38 AM
In her talk, Dr. Iva Hafner Bratkovič introduced the inflammasome, a part of our innate immune system that acts as a “potent inflammatory machine”. While these molecular complexes are essential for protecting us from pathogens, their dysregulation can contribute to a variety of diseases.
Read the full blog for the metaphor, methods, and implications: ctgct.si/en/objave/fi...
Comments and questions welcome. Specially on potential paths to in vivo testing and clinical translation.
@rjerala.bsky.social #CTGCT
Comments and questions welcome. Specially on potential paths to in vivo testing and clinical translation.
@rjerala.bsky.social #CTGCT
From Film Editing to RNA Editing: How RNA Trans-splicing Could Treat CTNNB1 Syndrome - CTGCT
Imagine you're in a movie editing studio, trying to fix a film with some corrupted scenes. The film is vital, as it plays a crucial role in the development and function of an entire movie franchise. S...
ctgct.si
September 26, 2025 at 12:41 PM
Read the full blog for the metaphor, methods, and implications: ctgct.si/en/objave/fi...
Comments and questions welcome. Specially on potential paths to in vivo testing and clinical translation.
@rjerala.bsky.social #CTGCT
Comments and questions welcome. Specially on potential paths to in vivo testing and clinical translation.
@rjerala.bsky.social #CTGCT
Why this matters: one PTM can potentially correct many different mutations across the gene; the correction happens under native gene control (reducing overexpression risk); and it can address problems where standard gene replacement is limited.
September 26, 2025 at 12:41 PM
Why this matters: one PTM can potentially correct many different mutations across the gene; the correction happens under native gene control (reducing overexpression risk); and it can address problems where standard gene replacement is limited.
Importantly, the team confirmed induced trans-splicing in the endogenous CTNNB1 transcript, not only in artificial reporters — a key step toward biological relevance.
September 26, 2025 at 12:41 PM
Importantly, the team confirmed induced trans-splicing in the endogenous CTNNB1 transcript, not only in artificial reporters — a key step toward biological relevance.
To identify effective PTMs they used a split-YFP reporter to screen candidates and tested modifications that improve yield — for example, elements that influence PTM expression and processing. These changes increased trans-splicing efficiency.
September 26, 2025 at 12:41 PM
To identify effective PTMs they used a split-YFP reporter to screen candidates and tested modifications that improve yield — for example, elements that influence PTM expression and processing. These changes increased trans-splicing efficiency.
Practically, researchers design a pre-trans-splicing molecule (PTM) that pairs with the target CTNNB1 pre-mRNA and provides a correct sequence to be spliced in. Think of the PTM as the spare film reel used to patch the damaged footage.
September 26, 2025 at 12:41 PM
Practically, researchers design a pre-trans-splicing molecule (PTM) that pairs with the target CTNNB1 pre-mRNA and provides a correct sequence to be spliced in. Think of the PTM as the spare film reel used to patch the damaged footage.
The paper explores RNA trans-splicing: an RNA-level editing strategy that replaces portions of a defective transcript with a healthy sequence, rather than altering DNA.
September 26, 2025 at 12:41 PM
The paper explores RNA trans-splicing: an RNA-level editing strategy that replaces portions of a defective transcript with a healthy sequence, rather than altering DNA.
CTNNB1 syndrome results from diverse, dispersed mutations in the CTNNB1 gene, which encodes β-catenin (a protein important for neurodevelopment) which makes single-target fixes impractical.
September 26, 2025 at 12:41 PM
CTNNB1 syndrome results from diverse, dispersed mutations in the CTNNB1 gene, which encodes β-catenin (a protein important for neurodevelopment) which makes single-target fixes impractical.
Congratulations to everyone involved in this great achievement!
September 25, 2025 at 10:45 AM
Congratulations to everyone involved in this great achievement!