Batten Disease – Treatment with Gene Therapy
Batten disease, also known as ceroid lipofuscinosis neuronal (CLN), is a group of rare neurological disorders caused by mutations in genes from the CLN family. These genetic conditions lead to the accumulation of harmful substances in cells, resulting in progressive neurological impairment.
Gene therapies for Batten disease aim to address the root cause of the disorder by:
- Restoring healthy gene expression through antisense oligonucleotides, RNA interference, or gene insertion.
- Correcting mutated sequences using advanced tools such as CRISPR base editors and CRISPR deletions.
Recent News on Gene Therapies for Batten Disease
- 11th October 2019: A groundbreaking proof of concept for personal RNA therapy targeting an ultra-rare form of Batten’s disease. Read the full study.
- 23rd October 2018: The first genome-based therapy for CLN7 Batten Disease was successfully introduced, marking a milestone in genetic treatments. Learn more here.
Current Experimental Genetic Treatments
- 2017 – Ongoing:
Milasen, a clinical trial for one person, uses antisense oligonucleotide therapy. This personalized approach was developed in response to a unique genetic mutation identified in a single patient.
Approved Genetic Treatments for Batten Disease
Currently, no fully approved genetic treatments exist for Batten disease. However, ongoing research and experimental therapies hold significant promise for future advancements.
Popular Articles on Genetic Innovations
- CRISPRi switches off 13 genes at once.
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Watch Gene Therapy in Action
- Batten Disease: A Family's Journey with Gene Therapy
- How Gene Therapy Works
- CRISPR Explained: Revolutionary Gene Editing
Random Quote
"Therefore, I have proposed the word ‘gene.’ The ‘gene’ is nothing but a very applicable little word. It may be useful as an expression for the ‘unit factors’… demonstrated by modern Mendelian researchers."
— Wilhelm Johannsen
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Genome Context continues to explore innovative therapies for Batten disease and other genetic conditions, shaping a brighter future for medicine and humanity. Join us on this transformative journey.