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CTGCT

@ctgct.bsky.social

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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.

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CTGCT @ctgct.bsky.social · 21h

We, the CTGCT, have joined the European statement to preserve the Hospital Exemption in the revised EU pharmaceutical legislation.

This statement reflects a united effort to ensure that Europe’s ATMP ecosystem remains both innovative and accessible.

ctgct.si/en/objave/ac...

Academic developers for affordable medicines - CTGCT

CTGCT (Centre for the Technologies of Gene and Cell Therapy, at National institute of Chemistry, Ljubljana) has joined the European statement to preserve the Hospital Exemption in the revised EU pharm...

CTGCT @ctgct.bsky.social · 23h

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

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 @ctgct.bsky.social · 23h

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.

CTGCT @ctgct.bsky.social · 23h

On Monday, the same day the #Nobel for Medicine was announce to be for research on #immunology and how it help develop #cancer treatments, we had a webinar from @ivahb.bsky.social from the Kemijski inštitut - National Institute of Chemistry title "Inflammasome-inspired cancer immunotherapy"

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 @ctgct.bsky.social · 3d

Check our latest post where we review the events of the CTGCT Days 2025

CTGCT DAYS 2025 – Spotlight on Tomorrow’s Medicine

ctgct.si/en/objave/sp...

CTGCT DAYS 2025 - Spotlight on Tomorrow’s Medicine - CTGCT

CTGCT Days 2025 gathered over 130 experts in Ljubljana for three days of cutting-edge discussions on immunotherapy, rare diseases, and gene editing, showcasing breakthroughs from global leaders alongs...

CTGCT @ctgct.bsky.social · 14d

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

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 @ctgct.bsky.social · 14d

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.

CTGCT @ctgct.bsky.social · 14d

Importantly, the team confirmed induced trans-splicing in the endogenous CTNNB1 transcript, not only in artificial reporters — a key step toward biological relevance.

CTGCT @ctgct.bsky.social · 14d

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.

CTGCT @ctgct.bsky.social · 14d

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.

CTGCT @ctgct.bsky.social · 14d

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.

CTGCT @ctgct.bsky.social · 14d

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.

CTGCT @ctgct.bsky.social · 14d

🎬 Imagine a film with corrupted scenes scattered throughout. Instead of fixing each scene, you replace the damaged parts at once with clean footage. That analogy frames our new blog:
From Film Editing to #RNA Editing: How RNA Trans-splicing Could Treat #CTNNB1 Syndrome

CTGCT @ctgct.bsky.social · 15d

Congratulations to everyone involved in this great achievement!

Reposted by CTGCT

UCL News @uclnews.bsky.social · 16d

A global clinical trial for a new Huntington’s disease treatment has posted positive results today, announced by trial sponsor uniQure and UCL scientists Prof Sarah Tabrizi and @profwild.bsky.social @uclqsion.bsky.social @uclbrainscience.bsky.social. Watch on BBC News at One 📺

CTGCT @ctgct.bsky.social · 16d

#CTGCT Science Talks are back!

This time we have a talk open to everyone by dr. Iva Hafner Bratkovič from the Department of Synthetic Biology and Immunology at the @kemijski.bsky.social.
Her talk is titled "Inflammasome-inspired cancer immunotherapy"

Join us! forms.office.com/e/KJTTsMyETk
#Webinar

Reposted by CTGCT

GeneH @genehproject.bsky.social · 22d

During #CTGCTDays2025, the GENE H Conference – Gene Therapy covered genome editing, biosafety, and neuro-focused applications. UC/GeneT presented SCA3 strategies (allele-specific silencing & gene inactivation). Program& abstracts: ctgct.si/en/ctgct-day...

@kemijski.bsky.social
@ctgct.bsky.social

CTGCT @ctgct.bsky.social · 29d

Unfortunately, it is fully booked, but stay tuned to see live updates during the #CTGCTDays2025 about these events and more. Don’t miss our next events!

Link to the conference page: ctgct.si/en/ctgct-day...

#Conference #GCT #Research

CTGCT Days 2025 - CTGCT

The CTGCT is organizing their 2025 conference. 3 days full of events which will take place in Ljubljana, Slovenia from the 15th to the 17th of September.

CTGCT @ctgct.bsky.social · 29d

We are looking forward to what we hope will become a forum where participants, attendees and sponsors will be able to learn more about the work being done in Gene and Cell Therapy and share new ideas that will spark future collaborations.

CTGCT Days 2025 - CTGCT

The CTGCT is organizing their 2025 conference. 3 days full of events which will take place in Ljubljana, Slovenia from the 15th to the 17th of September.

CTGCT @ctgct.bsky.social · 29d

And in parallel our #GoldSponsor accela.eu will have their Technologies for cell and gene therapies Workshops & Demonstrations.

ACCELA - Accelerate your biomedical research - Accela - Accelerate your biomedical research

Default Title Molecular technologies HTP end-point and qPCR SNP genotyping, Gene expression, Target enrichment, Single cell genomics, Digital PCR Show more cellular analysis Fluorescence, Luminescence...

CTGCT @ctgct.bsky.social · 29d

3 days left until the start of the #CTGCTDays2025!

3 days full of events:
CTGCT Conference – Cell Therapy
Gene conference – Gene Therapy
#RoundTable I – Regulatory and Clinical Pathways for Gene and Cell Therapy
#RoundTable II – Bridging Innovation and Commercialization in Gene and Cell Therapy

CTGCT @ctgct.bsky.social · 29d

#Kemomed, d.o.o. (Slovenia) is bringing solutions for all current and future research questions that researchers and people working in laboratories might have. They help you reach your goals easier and faster.

Kemomed is a Coffee Break #sponsor for the #CTGCTDays2025
#CTGCT
#GCT
#Conference

CTGCT @ctgct.bsky.social · Sep 10

On Wednesday the 17th they have brought a representative from Thermo Fisher to present more about the product they have available in Slovenia.

Get a chance to meet their representatives in Slovenia while discussing your needs for biopharma research and development.

#CTGCT #GCT #Conference

CTGCT @ctgct.bsky.social · Sep 10

#Omega is the #SilverSponsor of the #CTGCTDays2025. They are a Slovenian company that can help you with research, development, education in the field of chemical analytics and molecular biology and Ensure reliable and smooth operation of your equipment.

CTGCT @ctgct.bsky.social · Sep 5

#Labena is an international company which offers integrated solutions in laboratory and process analytics.
Labena Slovenia is a Coffee Break #sponsor for the #CTGCTDays2025. Get a chance to meet their representatives while discussing your needs for biopharma research during the CTGCT Days 2025.