If your enquiry is patient-related, please address the following questions (see also item 2 in the Q&A section below) on the contact form or by email to DCRT@LUMC.nl.

1. Who is the patient and what is their age?
2. What is the name of the disease and the gene that is involved?
3. Which tissues are affected by the disease? (we can currently only treat brain, spinal cord or eye)
4. What is the exact genetic mutation? (ask your clinician if you don’t know)
5. In which country are you based and where are you currently being treated?
6. Has the diagnosis been confirmed by a clinician?
7. May we contact the clinician for more information about your diagnosis?
8. Have you already discussed the possibility of a genetic treatment with someone? If so, with whom?
9. What would you like to gain from treatment?

If your enquiry is not patient-related, you may type in your question directly on the contact form below.

Q&A

1. Which diseases are eligible for RNA therapy at DCRT?

We focus on progressive genetic diseases that affect tissues of the brain, the spinal cord or the eye.

In this list, you will find the genes of interest that we have identified so far. The list is non-exhaustive so please contact us if there are other mutations that we are not aware of, or if you want to know more about RNA therapy. Feel free to send your questions to DCRT@LUMC.nl.

2. My child or relative has a genetic disease. Can RNA treatment help?

To determine whether the patient is eligible for the type of RNA treatment DCRT develops, we require additional information about the patient and the disease:

1. Who is the patient and what is their age?
2. What is the name of the disease and the gene that is involved?
3. Which tissues are affected by the disease? (we can currently only treat brain, spinal cord or eye)
4. What is the exact genetic mutation? (ask your clinician if you don’t know)
5. In which country are you based and where are you currently being treated?
6. Has the diagnosis been confirmed by a clinician?
7. May we contact the clinician for more information about your diagnosis?
8. Have you already discussed the possibility of a genetic treatment with someone? If so, with whom?
9. What would you like to gain from treatment?

You may send this information via e-mail to DCRT@LUMC.nl or via the contact form on top of this page.

Disclaimer: Unfortunately, for the majority of mutations, our RNA treatment is not an option. We are happy to look into your specific case, but please keep expectations low.

3. Where can I find more information about the RNA treatment that DCRT is working on?

On our webpage https://rnatherapy.nl/service you can read about our RNA treatment approach.

Furthermore, there are three interesting DCRT webinars on the development of n1 RNA therapies, which disease-causing variants are eligible for N=1 ASOs? and ASO-based therapies for retinal diseases that are published on the site of the Oligonucleotide Therapeutics Society.

Our talks, posters and publications can also be found on this website under the button with the same name.

4. How can I stay up to date with the work of DCRT?

You can follow us on LinkedIn to stay informed about the activities of the DCRT. Also, we have a news blog, where you can find the latest news updates. Or come and talk with us at one of the events where we will present!

5. Where can I find more information about DCRT?

On our webpage https://rnatherapy.nl/about us you will find more information about the DCRT researchers.

6. I want to make a donation. How can I do this?

Very nice that you want to support our work. Please e-mail us at DCRT@LUMC.nl so we can explore together if your donation should go to the DCRT in general or to projects or diseases we work on specifically.