ZNM – Zusammen Stark! e.V., an association dedicated to supporting patients with rare muscle diseases called centronuclear myopathies (CNM), is proud to announce the award of a grant of 81,400 euros for a ground-breaking research initiative led by Dr Haicui Wang of the Department of Human and Animal Cell Lines at the Leibniz Institute DSMZ-German Collection of Microorganisms and Cell Cultures GmbH. This major grant, funded entirely by donations, aims to advance potential treatments for CNM by creating multiple muscle models (each with a specific CNM mutation) and then treating them with CRISPR technology.
Translated with DeepL.com (free version)
Centronuclear myopathies are a group of rare muscle diseases caused by mutations in specific genes, including MTM1, DNM2, BIN1, RYR1 and TTN. The genetic diversity of individual ZNM patients poses major challenges to the development of a standardised therapeutic strategy. Current gene therapy approaches for CNM include the replacement of disease-causing genes or functional compensation with related genes. In this context, the use of CRISPR base editors opens up new possibilities for precise mutation correction.
3D CNM muscle model to test the efficacy and safety of CRISPR treatment for CNM patients
‘CRISPR gene editing tools hold great potential for the treatment of genetic diseases, including CNM. Therefore, this project aims to create and use a 3D human CNM muscle model to evaluate the efficacy and safety of precise mutation correction with CRISPR base editing before testing it on patients to accelerate its clinical application,’ emphasised Dr Wang, the scientist at Leibniz Institute DSMZ and leader of the new project. To achieve this, Dr Wang will create ten 3D CNM muscle models with mutations from four CNM genes: MTM1, DNM2, BIN1 and RYR1, study the impairment of muscle function and finally attempt to treat the mutant CNM muscle model by base editing in the form of mRNA.
Dr Wang’s innovative research aims to improve our understanding of the genetic basis of CNM and provide a platform for the development of potential therapies. It could also improve genotype-phenotype correlation, aiding future CNM diagnosis and mechanistic studies.
Hope for a better future in the community
Dr Holger Fischer, President of ZNM – Zusammen Stark!, expressed the organisation’s enthusiasm for supporting this project: ‘We are very pleased to support Dr Wang’s research initiative. CNM are rare diseases and finding effective treatments has been a challenge. Dr Wang’s project is an important step towards potential therapies for CNM patients. We are proud to support this groundbreaking work.’
This grant continues the commitment of ZNM – Zusammen Stark! to advance research and improve the lives of individuals and families affected by CNM. It is part of the broader mission of ZNM – Zusammen Stark! to connect those affected, support their families and invest in research that can make a difference.
Support our mission
We extend our heartfelt thanks to our donors, research supporters and external reviewers whose continued support has made this groundbreaking research possible. As we continue to advance our understanding and treatment of centronuclear myopathies, we invite you to continue to support our research through donations to find treatments for CNM patients.
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About centronuclear myopathies
Centronuclear myopathies are very rare muscle diseases. The most affected children with centronuclear myopathies cannot walk; they need a feeding tube and a ventilator to support their breathing. However, this does not prevent them from learning, playing and discovering the world like other children their age.
About ZNM – Zusammen Stark! e.V.
ZNM – Zusammen Stark! e. V. is a self-help organisation for myotubular myopathy and other centronuclear myopathies (CNM). We represent 258 people from 92 families with CNM in Germany, the Netherlands, Austria, Switzerland and Belgium. As a self-help organisation, our main goal is to connect those affected and their families and to support each other in everyday life. This is mainly done through our annual family conference, webinars and our closed Facebook support group. We also actively invest in research to find a treatment for these conditions. For more information, please visit www.znm-zusammenstark.org
About the Leibniz Institute DSMZ-German Collection of Microorganisms and Cell Cultures
The Leibniz Institute DSMZ – German Collection of Microorganisms and Cell Cultures GmbH is the world’s most diverse collection of biological resources (bacteria, archaea, protists, yeasts, fungi, bacteriophages, plant viruses, genomic bacterial DNA as well as human and animal cell cultures). Microorganisms and cell cultures are collected, researched and archived at the DSMZ. As an institution of the Leibniz Association, the DSMZ with its extensive scientific services and biological resources has been a global partner for research, science and industry since 1969. The DSMZ is recognised as a non-profit organisation, the first registered collection in Europe (Regulation (EU) No. 511/2014) and certified according to the ISO 9001:2015 quality standard. As a patent depository, it offers the only possibility in Germany to deposit biological material in accordance with the requirements of the Budapest Treaty. In addition to scientific services, research is the second pillar of the DSMZ. The institute, based on the Science Campus Braunschweig-Süd, houses more than 85,000 cultures and biomaterials and has around 220 employees. www.dsmz.de
Judith Wartenburger
Assistant to the board of
ZNM – Together Strong! e.V
Tel.: 01515 4820534
Email: judith.wartenburger@znm-zusammenstark.org
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